The association alfa-enolase/plasmin is a new selective target for treating muscular pathologies

The association alfa-enolase/plasmin is a new selective target for treating muscular pathologies: Researchers at the Bellvitge Biomedical Research Institute (IDIBELL) have described a new selective target in muscle regeneration. This is the association of alpha-enolase protein and plasmin. The finding could be used to develop new treatments to regenerate muscular injuries or dystrophies. The study has been published in PLOS ONE journal

Dantrolene may help combat Duchenne muscular dystrophy in boys

Dantrolene may help combat Duchenne muscular dystrophy in boys: Drugs are currently being tested that show promise in treating patients with Duchenne muscular dystrophy (DMD), an inherited disease that affects about one in 3,600 boys and results in muscle degeneration and, eventually, death.
Now, scientists at UCLA have found a drug, already approved by the U.S. Food &Drug Administration and being used in humans, that provides a powerful boost to the therapy currently being tested in clinical trials. They hope this one-two punch used in combination will overcome the genetic mutations that cause DMD, restore a missing protein needed for proper muscle function and allow those affected by the disease to lead relatively normal lives.

Injecting Wnt7a protein could prevent Duchenne muscular dystrophy

Injecting Wnt7a protein could prevent Duchenne muscular dystrophy: Scientists have discovered that injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, findings that could lead to a therapy akin to the use of insulin by diabetics. These results were published today in the Proceedings of the National Academy of Sciences by Dr. Julia von Maltzahn and Dr. Michael Rudnicki, the Ottawa scientist who discovered muscle stem cells in adults.

Great news from Holyrood

Great news from the debate results from Holyrood

The meeting last night at Holyrood was a great success. The Public
Health Minister, Michael Matheson, advised that £16 million will be
allocated for wheelchair services, £1.5 million for respiratory
services and finally although no specific amount was stated he said
funding will be given into research for Duchenne! This is a fantastic
result which has been a long time coming!

John Miller, grandparent to Lee, a great friend of Action Duchenne's
worked tirelessly to get this debate to happen and we are very
grateful for his wonderful efforts.

Summit Corporation PLC Press Release - Repeat Dosing of SMT C1100 for DMD meets Endpoints in phase 1 trial - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Summit Corporation PLC Press Release - Repeat Dosing of SMT C1100 for DMD meets Endpoints in phase 1 trial - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Oxford, UK, 7 November 2012 - Summit (AIM: SUMM), a UK drug discovery company,
announces that the repeat dosing of the utrophin upregulator SMT C1100 for the
treatment of the fatal muscle-wasting disease Duchenne Muscular Dystrophy
('DMD') has successfully met the endpoints as part of a Phase 1 clinical trial
in healthy volunteers. The trial evaluated a new formulation of SMT C1100 and
the results showed that upon repeat dosing, concentrations of the drug achieved
in the blood plasma, stabilised at levels that from preclinical studies are
expected to significantly increase utrophin protein production. The new
formulation was also shown to be safe and well-tolerated in this Phase 1 trial.

HCT 1026 may help stem debilitating effects of muscular dystrophy

HCT 1026 may help stem debilitating effects of muscular dystrophy: Cedars-Sinai Heart Institute researchers have found that an experimental compound may help stem the debilitating effects of muscular dystrophy by restoring normal blood flow to muscles affected by the genetic disorder.

The researchers studied HCT 1026, a new type of molecule in which nitric oxide is chemically attached to a standard anti-inflammatory agent, in a preclinical model of muscular dystrophy. Results suggest HCT 1026 may be beneficial for the treatment of Duchenne muscular dystrophy, which begins in early childhood, and Becker muscular dystrophy, which often occurs later in adulthood.

New Ataluren UK Clinical Trials - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

New Ataluren UK Clinical Trials - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: We are delighted to inform you that PTC Therapeutics has been able to establish an extension study in the UK for their drug ataluren for patients who participated in the Phase 2b. This extension study will take place at two centres: Newcastle and London. The background to this decision by PTC Therapeutics can be found at http://www.actionduchenne.org/viewarticle?news=387 .
Secondly, PTC Therapeutics is in active communication with European regulators to run a wider confirmatory study with the aim of confirming the findings of the previous study. We look forward to welcoming PTC Therapeutics to our forthcoming annual conference where we hope there will be an opportunity to find out more about their plans.

First approval for AAV vector gene therapy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

First approval for AAV vector gene therapy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Netherlands biotech company UniQure have announced that Glybera has become the first gene therapy approved by regulatory authorities in the Western world.

It is the first medication approved for patients with rare metabolic disorder Lipoprotein Lipase Deficiency and commercial roll-out to begin second half of 2013. The company that has taken this product over from AMT state that it now validates uniQure’s unique AAV-based gene therapy platform.

Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Prosensa has also received orphan drug designation by the European Commission for PRO045 and PRO053. These compounds are designed to skip exons 45 and 53 of the dystrophin gene and are expected to enter clinical development within the next 6 months.

In A First, An Experimental Drug May Help Boys With Muscular Dystrophy - Forbes

In A First, An Experimental Drug May Help Boys With Muscular Dystrophy - Forbes: A new experimental drug appears to improve the muscle function of boys with muscular dystrophy, increasing the distance the boys can walk in six minutes compared to when they began taking it a year before.

DMD: Reformulated Utrophin Upregulator Looks Good in Healthy Volunteers | Quest Magazine Online

DMD: Reformulated Utrophin Upregulator Looks Good in Healthy Volunteers | Quest Magazine Online: Summit PLC, a drug discovery company based in Oxford, U.K., has announced that SMT C1100, its experimental oral drug for Duchenne muscular dystrophy (DMD), appears safe and well-tolerated in healthy volunteers at all doses tested, and that it reaches levels in the bloodstream believed to be adequate for a therapeutic benefit.

Positive Phase 1 trial results for SMT C1100 (Summit PLC) - PPMD Community

Positive Phase 1 trial results for SMT C1100 (Summit PLC) - PPMD Community: Following closely on the heels of the good news about Sarepta’s exon 51 skipping strategy, PPMD is pleased to report that Summit PLC�has been able to achieve sufficient plasma levels of its reformulated utrophin-upregulating drug to justify a phase I/II trial in Duchenne (Read Summit's Press Release). At PPMD we have always believed in “hedging our bets” and investing widely in different therapies, some of which are mutation specific and some of which are not. Unlike exon-skipping strategies, upregulating utrophin is not a mutation specific strategy and, if effective, could potentially benefit anyone with Duchenne

Promising News from Sarepta on Exon 51 Skipping Trial - PPMD Community

Promising News from Sarepta on Exon 51 Skipping Trial - PPMD Community: The much-anticipated 48-week results on eteplirsen, a drug designed to skip exon 51, were presented today by Sarepta Therapeutics�on a webinar and press release. On the webinar, Sarepta’s Chief Executive Chris Garabedian presented data about the dystrophin found in study participants’ muscle biopsies and the 6-minute walk results.

UCLA scientists awarded $8.7 million in grants for stem cell research - Los Angeles health | Examiner.com

UCLA scientists awarded $8.7 million in grants for stem cell research - Los Angeles health | Examiner.com: UCLA stem cell researchers are on the cutting edge of developing new technology in their field. On September 6, UCLA Health System announced that three researchers had been awarded a total of $8.7 million in basic biology grants from the state stem cell agency.

The flu vaccine and Duchenne: What you need to know - PPMD Community

The flu vaccine and Duchenne: What you need to know - PPMD Community: The CDC (Center for Disease Control and Prevention) has joined with The� American Academy of Pediatrics, Families Fighting Flu and Family Voices to spread the message about the importance of�influenza vaccination�to protect our children.
�
The influenza vaccine, or “flu shot,” is an injection into the muscle, usually given in the upper arm or thigh. The flu shot contains inactivated (dead) virus and is recommended for patients with neuromuscular diseases and patients taking chronic steroids. Because the virus is dead, there is no risk of getting the flu from the flu shot. (The “nasal spray flu vaccine” contains living virus, and is NOT recommended for people with�neuromuscular diseases.)�

Beyond Exon 51 - PPMD Community

Beyond Exon 51 - PPMD Community: As Sarepta (formely AVI) announces promising results from its phase II interim data�and GSK follows up its promising phase II data with a phase III study, things are looking good for an exon-skipping based therapy for Duchenne…if you happen to be in the 13% of those with Duchenne who have a mutation that can be improved by skipping exon 51. For quick reference, this group includes out-of-frame deletions in exons 45-50, 47-50, 48-50, 49-50, 50, and 52.

Father kayaks across the English Channel to raise funds to fight son’s illness - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Father kayaks across the English Channel to raise funds to fight son’s illness - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Father from Dibden Purlieu, Stuart Littler, is to kayak across the English channel with his two friends, Charlie Brett and Jon Turner to raise funds to find viable treatments for his son, Ben. Ben, who is 6 in September, was diagnosed with Duchenne Muscular Dystrophy in December 2009. Since then the family have been working with charity Action Duchenne to raise money to improve awareness and funds for this life limiting condition. The three friends are novice kayakers and have never attempted anything like this before. In the process they have already met their initial target of raising �5000, and are now aiming for �10,000, to be donated to Action Duchenne. The crossing is set to take place during week commencing 27 August, and will be dependent on tides and weather.

Sun News : New research shows hope of cure for muscular dystrophy

Sun News : New research shows hope of cure for muscular dystrophy: EDMONTON -- There is hope in sight for those suffering from muscular dystrophy after a breakthrough discovery at the University of Alberta that could someday cure the disease.

Dr. Toshifumi Yokota, a researcher at the university's faculty of medicine and dentistry, has been working on a gene treatment therapy for over five years that targets the gene responsible for causing Duchenne muscular dystrophy.

Progress on Duchenne muscular dystrophy via antisense exon region skipping

Progress on Duchenne muscular dystrophy via antisense exon region skipping: new work by a Japanese team that shows a promising approach in an animal model of Duchenne muscular dystrophy, the most common form of the disease.

Ryan Lochte - End Duchenne: Ryan Lochte

Ryan Lochte - End Duchenne: Ryan Lochte: Olympic Champion Ryan Lochte lost a member of his family to Duchenne muscular dystrophy, a fatal disease that robs boys of their muscle movement. Ryan is dedicated to the fight to end Duchenne and hopes you will join him.

John Miller speaks to the Sunday Post about DMD and his hopes for the recent steps forward in research - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

John Miller speaks to the Sunday Post about DMD and his hopes for the recent steps forward in research - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Parents aren’t meant to outlive their children. So we can’t imagine what it must be like for a grandparent having to contemplate living longer than their grandchild. But that’s exactly the position in which Edinburgh’s John Miller finds himself. John’s grandson Lee, 14, has Duchenne Muscular Dystrophy.

Resveratrol decreases inflammation and increases utrophin gene expression in the mdx mouse model of duchenne muscular dystrophy

Resveratrol decreases inflammation and increases utrophin gene expression in the mdx mouse model of duchenne muscular dystrophy: Duchenne muscular dystrophy (DMD) is a lethal genetic disease with no cure. Reducing inflammation or increasing utrophin expression can alleviate DMD pathology. Resveratrol can reduce inflammation and activate the utrophin promoter. The aims of this study were to identify an active dose of resveratrol in mdx mice and examine if this dose decreased inflammation and increased utrophin expression.

Ataluren Update - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Ataluren Update - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: The pivotal Phase 2b trial of ataluren, an investigational new drug being studied in nonsense mutation Duchenne/Becker muscular dystrophy (DBMD), was completed in late 2009. It had enrolled 174 patients at 37 trial sites in 11 countries on four continents. As PTC and statistics experts have analyzed the complex data from this trial using various statistical methods, the company has come to understand that the results are very promising.

Action Alert! Contact your Senators to help save vital Duchenne programs at the CDC - PPMD Community

Action Alert! Contact your Senators to help save vital Duchenne programs at the CDC - PPMD Community: Take Action: Contact your Senators
In May we asked everyone to reach out to their member in the House of Representatives on this issue.
Now it's time to contact your two Senators!�Deadline: Friday, July 13
�



PPMD, along with other stakeholders in the disability community, are urging our advocates to contact their Congressional representatives to ask that they sign on to a Dear Colleague letter being circulated bySenator Roger Wicker (R-MS) and Senator Frank Lautenberg (D-NJ) that expresses concern to CDC Director Tom Frieden about the proposed consolidation of the NCBDDD.

Thanks in part to advocacy efforts by the disability community last year, the proposed consolidation was stopped.� We are asking for your support this year to achieve a similar result.

Action Alert! Contact your Senators to help save vital Duchenne programs at the CDC - PPMD Community

Action Alert! Contact your Senators to help save vital Duchenne programs at the CDC - PPMD Community: We need your voice to help save critical Duchenne related programs at the Center for Disease Control (CDC)!

Deadline: Friday, July 13
�
The Administration's Fiscal Year 2013 budget request again proposes to consolidate muscular dystrophy and other programs within the CDC's National Center for Birth Defects and Developmental Disabilities (NCBDDD) and to eliminate the funding level for the center. The work of the NCBDDD is directly responsible for much of the progress made over the last decade to improve care of those with Duchenne. Specifically, the development and issuance of the Care Considerations have increased the life expectancy by approximately 10 years, a remarkable improvement.

Landmark FDA legislation becomes law - PPMD Community

Landmark FDA legislation becomes law - PPMD Community: Major win for the Duchenne community
PPMD applauds the President for signing into law the Prescription Drug User Fee Act also known as�The Food and Drug Administration Safety and Innovation Act.�Most recently, PPMD worked with the Everylife Foundation for Rare Diseases and Genetic Alliance to organize a letter to Congress�signed by nearly 120 patient advocacy organizations urging the inclusion of specific provisions within the final legislation. PPMD is grateful for the many actions advocates have taken to achieve this success. We also appreciate our board’s clear and compelling position on FDA engagement issues that produced a�board policy�and guided our advocacy efforts.
�
This is a major win for the Duchenne community as well as the rare disease community as a whole.

The legislation includes:
A provision from the Senate bill that would deepen patient engagement in the review of medical products.
Content from the House bill that would provide explicit direction to FDA in developing Fast Track and Accelerated Review guidance that recognizes the small patient populations.
A provision from the House bill that would incent industry to develop treatments for pediatric rare diseases by providing a voucher the sponsor could use to speed up FDA review of another candidate treatment.

Read-through molecule improves muscle function in DMD

Read-through molecule improves muscle function in DMD: A compound that results in a read-through on nonsense mutations in messenger RNA (mRNA) restores dystrophin expression in an animal model of Duchenne muscular dystrophy (DMD), research shows.

Novel gene therapy approach may treat dysferlinopathies

Novel gene therapy approach may treat dysferlinopathies: The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's Hospital study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies.

Action Duchenne announces �5million skipDuchenne research fund to focus on delivering a cure for Duchenne Muscular Dystrophy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne announces �5million skipDuchenne research fund to focus on delivering a cure for Duchenne Muscular Dystrophy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: ACTION DUCHENNE ANNOUNCES �5MILLION SKIPDUCHENNE RESEARCH FUND TO FOCUS ON DELIVERING A CURE FOR DUCHENNE MUSCULAR DYSTROPHY

PTC Therapeutics initiates open-label study for Atlauren - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

PTC Therapeutics initiates open-label study for Atlauren - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: PTC Therapeutics, Inc. (PTC) today announced the initiation of an open-label study in the European Union, Israel, Australia and Canada for patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who received ataluren in a prior, PTC-sponsored clinical study. The primary objective of this study is to gain further information on the long-term safety and tolerability of ataluren, an investigational new drug. PTC launched a similar study in the United States in November 2010.

New compound could treat certain types of genetic disorders in muscles

New compound could treat certain types of genetic disorders in muscles: Scientists at UCLA have identified a new compound that could treat certain types of genetic disorders in muscles. It is a big first step in what they hope will lead to human clinical trials for Duchenne muscular dystrophy.

Duchenne muscular dystrophy, or DMD, is a degenerative muscle disease that affects boys almost exclusively. It involves the progressive degeneration of voluntary and cardiac muscles, severely limiting the life span of sufferers.

In a new study, senior author Carmen Bertoni, an assistant professor in the UCLA Department of Neurology, first author Refik Kayali, a postgraduate fellow in Bertoni's lab, and their colleagues demonstrate the efficacy of a new compound known as RTC13, which suppresses so-called "nonsense" mutations in a mouse model of DMD.

Phrixus Pharmaceuticals, Inc. Announces $623,000 in NIH Funding for Its Program in Duchenne Muscular Dystrophy

Phrixus Pharmaceuticals, Inc. Announces $623,000 in NIH Funding for Its Program in Duchenne Muscular Dystrophy: Phrixus Pharmaceuticals, Inc., a clinical-stage, specialty pharmaceutical company focused on innovative therapies for Duchenne muscular dystrophy (DMD) and heart failure, today announced that it has received an SBIR Phase 1 award for $623,000 from the National Institutes of Health (NIH) for its grant application titled "Treatment of Muscular Dystrophy-Associated Dilated Cardiomyopathy with P-188."
"This funding constitutes validation for the potential utility of Carmeseal in DMD. We expect that it will allow us to extend administration of Carmeseal to a new, clinically relevant route of administration, subcutaneous delivery," said Thomas A. Collet, president and CEO.

Arbroath Brother has lost both his soul mates - Local Headlines - Arbroath Herald

Brother has lost both his soul mates - Local Headlines - Arbroath Herald: AN ARBROATH couple have suffered the agony of losing a second son in as many years to a debilitating disease.

John Mathieson, who was just 21 years old, died on Tuesday afternoon.

Norman and Yvonne Mathieson, McGregor’s Walk, have worked tirelessly over the years to provide a better quality of life for their three sons, all of whom suffered from Duchenne muscular dystrophy.

Carl ‘Action’ Tilson to carry Olympic Flame on to stage at Olympic Torch Party, Albert Square Manchester - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Carl ‘Action’ Tilson to carry Olympic Flame on to stage at Olympic Torch Party, Albert Square Manchester - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: CARL ‘ACTION’ TILSON TO CARRY OLYMPIC FLAME ON TO STAGE AT OLYMPIC TORCH PARTY, ALBERT SQUARE MANCHESTER
Olympic torch bearer happy ending for Carl, thanks to help from volunteers at the torch bearer hotline

AVI BioPharma and PPMD team up to run the Rock 'n' Roll Seattle Marathon - PPMD Community

AVI BioPharma and PPMD team up to run the Rock 'n' Roll Seattle Marathon - PPMD Community: Run For Our Sons�(PPMD's endurance program) is teaming up with AVI BioPharma�in Saturday's Rock 'n' Roll Seattle Marathon�to raise money and awareness to help end Duchenne!
�
AVI and PPMD have a long history of working together to develop treatments that may help to prolong the lives of those living with Duchenne. We have always been grateful to the dedication AVI has shown to the Duchenne community as they continue to work on treatments that will help our sons live longer, stronger lives. Now they are taking that dedication a step further by lacing up their running shoes and pounding the streets of Seattle to help raise awareness and end Duchenne.

PPMD Leads Critical Letter for Rare Disease Community - PPMD Community

PPMD Leads Critical Letter for Rare Disease Community - PPMD Community: Parent Project Muscular Dystrophy (PPMD) has teamed up with�EveryLife Foundation for Rare Diseases�and�Genetic Alliance�to galvanize the rare disease community and send a strong message to Congress to ensure the most important provisions for the rare disease community were included in the final�Prescription Drug User Fee Act (PDUFA) legislation. The provisions are aimed at accelerating Food and Drug Administration (FDA) review of therapies to treat rare diseases and to enable FDA to more aggressively engage external experts in reviewing candidate therapies.

New Cardiomyopathy Study for DMD Recruiting in Ohio

New Cardiomyopathy Study for DMD Recruiting in Ohio: "Inclusion Criteria:

Males with Duchenne
Age 7 years or older
No signs of cardiomyopathy (preserved left ventricular systolic function)"

This study is taking place at two sites in Ohio: The Ohio State University (OSU) in Columbus and Cincinnati Children's Hospital Medical Center (CCHMC). 


Approximately 40 males with Duchenne will be enrolled in this study.

'via Blog this'

Summit Corporation PLC: Summit achieves SMT C1100 Phase 1 dosing milestone that triggers milestone payment

Summit Corporation PLC: Summit achieves SMT C1100 Phase 1 dosing milestone that triggers milestone payment: "SUMMIT ACHIEVES SMT C1100 PHASE 1 DOSING MILESTONE THAT TRIGGERS MILESTONE PAYMENT
* Trial moves to Multiple Dose stage
* Summit to Participate in Upcoming Industry Events
Summit (AIM: SUMM), a UK drug discovery company, today announced that it has successfully passed a milestone in the Phase 1 trial of SMT C1100 for the treatment of the fatal genetic disease Duchenne Muscular Dystrophy ('DMD'), which triggered the final payment from a $1.5 million funding agreement with US-based DMD organisations."

'via Blog this'

Boys to Men Campaign in Scotland - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Boys to Men Campaign in Scotland - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: What a brilliant day at the Scottish Parliament. Over 30 family members, those living with Duchenne Muscular Dystrophy and supporters attended a lobby day at the Scottish Assembly on Wednesday 6th June to launch Action Duchenne's Boys to Men Campaign.

MSP's agreed to follow up with a debate in Parliament and Scottish families are arranging a meeting with Health Minister Michael Matheson MSP. 

Please get involved in our Boys to Men Campaign by writing to your MP and coming to Westminster on Wednesday 

All Sites Now Recruiting for GSK Exon 51 Skipping Trial

All Sites Now Recruiting for GSK Exon 51 Skipping Trial: The purpose of this study is to determine if GSK2402968 is effective in the treatment of ambulant boys with DMD resulting from a mutation thought to be corrected by exon 51 skipping. Two doses of GSK2402968 and placebo will be used in this study. This study is sponsored by GlaxoSmithKline (GSK).

This study will be conducted in the United States only and is now recruiting at 14 different sites in the following cities: Sacramento CA; Stanford, CA; Pensacola, FL; Iowa City, IA; Kansas City, KS; Baltimore, MD; Minneapolis, MN; St. Louis, MO; New York City, NY; Durham, NC; Cincinnati, OH; Columbus, OH; Portland, OR and Dallas TX.

Webinar: New approaches to treating inflammation in Duchenne - PPMD Community

Webinar: New approaches to treating inflammation in Duchenne - PPMD Community: Parent Project Muscular Dystrophy�welcomes Dr. Michael Jirousek from�Catabasis�as our guest for a webinar on June 6, 2012 at 1pm eastern.*
�
Topic:�New approaches to treating inflammation in Duchenne
When:�Wednesday,�June 6, 1pm eastern
�
Inflammation is a key pathway in the disease pathway in Duchenne’s muscular dystrophy. CAT-1004 is a novel, anti-inflammatory agent being developed by Catabasis as a potential treatment for patients with Duchenne. This presentation will describe the science and rationale behind the development program.

Summit Corporation PLC : Summit Announces Initiation of Phase 1 Trial for the Treatment of Duchenne Muscular Dystrophy

Summit Corporation PLC : Summit Announces Initiation of Phase 1 Trial for the Treatment of Duchenne Muscular Dystrophy: OXFORD, UNITED KINGDOM--(Marketwire - May 25, 2012) - Summit (AIM: SUMM), a UK drug discovery company, today announced that it has dosed the first cohort of patients in a Phase 1 study of SMT C1100 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal, rare genetic disease characterized by rapidly worsening muscle weakness. SMT C1100, an oral small molecule compound, is a potential disease-modifying drug that works to increase, or upregulate, the amount of a naturally occurring protein called utrophin.

Tough Mudder completed!

On 12 May Ryan & Ettienne completed their tough mudder challenge and have surpassed their fundraising target for Logan - many thanks guys.

I will post photos as soon as I get them.

Opportunity for Scottish families - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Opportunity for Scottish families - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: The Muscle Help Foundation is happy to announce that the charity's 2012 flagship Games Inspired Muscle Dreams programme is now live with the online nomination process officially open! This compelling project offers a once-in-a-lifetime opportunity for children and young people from across the UK with Muscular Dystrophy to witness the power and inspiration of the 2012 Paralympic Games.

Action Duchenne trustee and supporter Mary Down becomes an Olympic torchbearer - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne trustee and supporter Mary Down becomes an Olympic torchbearer - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: On Friday 25th May, Mary Down, Action Duchenne trustee, supporter and grandmother will be carrying the Olympic Torch in Cardiff at about 17:40.�

Mary, who will be keeping her torch said that it is her ambition that all her grandchildren, including Seth will be able to bring out the torch in years to come when the Olympics returns to the UK.

Mary's grandson Seth has Duchenne Muscular Dystrophy and she was nominated by her son John for the amazing charity work she does.

PPMD endorses Senate version of Prescription Drug User Fee Act (PDUFA) after patient voice strengthened - PPMD Community

PPMD endorses Senate version of Prescription Drug User Fee Act (PDUFA) after patient voice strengthened - PPMD Community: As the U.S. Senate continues debate on legislation that seeks to accelerate delivery of safe and effective therapies to patients and to increase the patient voice as part of the therapy review process, PPMD�is pleased to offer our enthusiastic endorsement of the legislation.
�
Over the past several months, PPMD�has fought to include several key provisions included in the Senate version of the Food and Drug Administration Safety and Innovation Act, commonly referred to as the Prescription Drug User Fee Act or PDUFA.�A number of similar provisions are also included in a House bill expected to be voted on by that chamber next week.
�
The provisions included in the Senate user fee bill will help expand the patient voice when key decisions are being made, an issue of grave importance to our community and to the larger rare disease community.�The provisions will also provide the FDA with additional tools to more quickly review potential therapies for life-threatening conditions, including Duchenne.

Action Duchenne Scottish Parliament Lobby

Busy gathering my thoughts in preparation for the Action Duchenne 2012 lobby of the Scottish Parliament in a couple of weeks.

Points to be presented this year include:

- Provision of DEXA Scan services - section only was offered again but we have opted for a fully body scan to ensure full and properly comparable results are recorded. Note: partial scan results received at our last visit were not comparable with those from the full body scan at Logan's first appointment and so we will be insisting on full body scans from now on. How widespread is the offering of partial scans and are parent aware of their shortcomings?
- Relative scarcity of information and advice received from UK teams when compared to that provided to a friend in the US. DMD is a devastating and progressive disease and it is difficult enough just dealing with it, never mind having to constantly push for care, information and advice including scans, supplements, the use of antioxidants and so on.

Lots to do . . . .

Super League’s Supermen feature Andy Johnson and the Joining Jack campaign

Super League’s Supermen feature Andy Johnson and the Joining Jack campaign - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Former rugby league player Andy Johnson and father to Jack aged 4, is to feature in an hour long feature to be aired this week on Sky Sports. Andy, who played for Salford City Reds, Castleford Tigers, London Broncos, Huddersfield Giants and Wigan Warriors in the European Super League has set up Joining Jack, a charity to help raise awareness of Duchenne and to raise funds to help find a cure. Filmed just last week, the programme Super League’s Supermen will broadcast on Sky Sports 4 on Thursday 24 May, at 7pm.

Action Duchenne donates �225,000 to Halo Therapeutics

Action Duchenne donates �225,000 to Halo Therapeutics - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Action Duchenne is proud to announce that we have agreed to support Halo Theapeutics in their quest to develop HT-100, the company’s proprietary formulation of halofuginone, a derivative of a compound found in a plant used in Chinese herbal medicine. HT-100 has great potential and may be able to halt or even reverse fibrosis, which is the scarring that occurs in muscles of Duchenne boys, inhibiting inflammation that leads to muscle damage, and promoting health muscle regeneration.

Action Duchenne wins Good Cause Award!! - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne wins Good Cause Award!! - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Action Duchenne receives Good Cause Award from Bettys Tea Room, York. The Good Cause Award is a community grant of �500 awarded on the basis of supporting local families. We would like to thank the team at Bettys for their fantastic support.

Study identifies how skeletal muscle stem cells respond to muscle injury

Study identifies how skeletal muscle stem cells respond to muscle injury: A study conducted by Children's Hospital & Research Center Oakland scientists identifies how skeletal muscle stem cells respond to muscle injury and may be stimulated to improve muscle repair in Duchenne Muscular Dystrophy, a severe inherited disease of muscle that causes weakness, disability and, ultimately, heart and respiratory failure.

U of M researchers develop new muscular dystrophy treatment approach using human stem cells | Science Codex

U of M researchers develop new muscular dystrophy treatment approach using human stem cells | Science Codex: The research, published today in Cell Stem Cell, outlines the strategy for the development of a rapidly dividing population of skeletal myogenic progenitor cells (muscle-forming cells) derived from induced pluripotent (iPS) cells. iPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos.

This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy.

PPMD Holds Landmark Meeting with FDA Leaders to Address Trial Design, Endpoint,... -- HACKENSACK, N.J., May 3, 2012 /PRNewswire-USNewswire/ --

PPMD Holds Landmark Meeting with FDA Leaders to Address Trial Design, Endpoint,... -- HACKENSACK, N.J., May 3, 2012 /PRNewswire-USNewswire/ --: "The recent PPMD-FDA leadership meeting provides a solid foundation upon which we can build to develop and implement the tools and processes necessary to expedite the FDA's review of potential treatments and accelerate the delivery of safe and effective treatments to our patients," Furlong said.
"FDA leaders provided valuable information and perspectives on trial design and surrogate endpoint acceptance, two critically important issues, and expressed a desire to continue the discussion with industry leaders. PPMD looks forward to working with FDA to convene a larger roundtable discussion and other follow-up activities on these issues in the coming months, and will work with all patients and Duchenne stakeholders to ensure the community's voice is well-represented at these events."

Results from DuchenneConnect Study - PPMD Webinar Follow up - PPMD Community

Results from DuchenneConnect Study - PPMD Webinar Follow up - PPMD Community: Please see the list of Q&A's following the webinar video below, and�let me know if you have any additional follow up questions!

End of season Score4Duchenne Support - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

End of season Score4Duchenne Support - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Some of you may recall that Dundee FC Supporters Society kindly helped to support our Score4Duchenne appeal on Boxing Day.Today is the final day of the season and to celebrate and thank everyone involved with Dundee FC Supporters Society Community Project Action Duchenne will join other charities today at Dens Park.

At half time each of the charities from throughout the season will go onto the pitch bearing their own flag, kindly sponsored by Dundee FC, led by the Fintry Pipe Band to visually show the many good causes hosted this season and raise further awareness for the charities.

MARTIN BASHIR, the TV journalist, explains why he’s backing the search for a cure for Duchenne muscular dystrophy.

Martin has become patron of Action Duchenne, the UK muscular dystrophy charity which campaigns for a cure and improved treatments for the cruel muscle-wasting condition which claimed the life of his brother Tommy whom he describes as “the most influential person in my life”.

Summit announces approval for Phase 1 Clinical trial - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Summit announces approval for Phase 1 Clinical trial - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Summit Corporation plc (‘Summit’ or ‘the Company’) SUMMIT RECEIVES REGULATORY APPROVAL TO ENTER SMT C1100 INTO A PHASE I CLINICAL TRIAL Oxford, UK, 24 April 2012, Summit (AIM: SUMM), a UK drug discovery company, today announces that its Phase I Clinical Trial Application (‘CTA’) for SMT C1100 has been approved by the UK regulatory body, the Medicines and Healthcare products Regulatory Agency (‘MHRA’).

Action Duchenne supports ground-breaking project at the Royal Holloway, University of London - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne supports ground-breaking project at the Royal Holloway, University of London - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Action Duchenne is to provide �160,000 of funding to support a pioneering project at Royal Holloway, University of London (RHUL). The project, which is being led by Dr Keith Foster and Prof. George Dickson in the School of Biological Sciences at Royal Holloway, is looking at a system to produce full length dystrophin. Dystrophin is the protein that protects and builds muscle, and it is the lack of dystrophin that causes muscular dystrophy. The system has shown dystrophin expressed at the cell membrane which is an indication that it has a therapeutic effect.

Well done to all Action Duchenne London Marathon Runners - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Well done to all Action Duchenne London Marathon Runners - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Congratulations to the 13 people who took part in the London Marathon on Sunday in support of Action Duchenne. A hotter day than expected made conditions challenging for the 35,000 people on the 26.2 mile course but we are happy to report that all Action Duchenne runners looked like they were coping admirably. Well done everyone - please get in contact and let us know how you got on!

If yo'veu been inspired to take part in the London Marathon next year the ballot for places opens on Monday the 30th April, and will fill up within a day. The organisers will contact you by October to let you know if you have been successful. Email kate@actionduchenne.org to register an interest and we will sent you the ballot information.

Action Duchenne supports ground-breaking project at the Royal Holloway, University of London - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne supports ground-breaking project at the Royal Holloway, University of London - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Action Duchenne is to provide �160,000 of funding to support a pioneering project at Royal Holloway, University of London (RHUL). The project, which is being led by Dr Keith Foster and Prof. George Dickson in the School of Biological Sciences at Royal Holloway, is looking at a system to produce full length dystrophin. Dystrophin is the protein that protects and builds muscle, and it is the lack of dystrophin that causes muscular dystrophy. The system has shown dystrophin expressed at the cell membrane which is an indication that it has a therapeutic effect.

Fling for Logan

On Saturday 12 May 2012 Ryan Linton and Ettienne Viljoen will be undertaking the Tough Mudder challenge to help us to raise funds for Logan.

Tough Mudder events are hardcore 10-12 mile obstacle course with 25 plus obstacles designed by the Special Forces to test your all around strength, stamina, mental grit, and camaraderie. As the leading company in the booming obstacle course industry, Tough Mudder has already challenged half a million inspiring participants worldwide. But Tough Mudder is more than an event, it’s a way of thinking.

Ryan and Ettienne will be putting themselves through this grueling challenge at Boughton House and Estate, Kettering Northhamptonshire.

http://www.toeandheel.com/flingforlogan/other_tough_mudder.php for details

AVI BioPharma - News Release

AVI BioPharma - News Release: AVI BioPharma to Present Additional Data From the Phase IIb Study of Eteplirsen for the Treatment of Duchenne Muscular Dystrophy at the 2012 AAN Annual Meeting.Findings will be presented today at the American Academy of Neurology 64th Annual Meeting in New Orleans, Louisiana. Principal investigator, Jerry R. Mendell, M.D. of Nationwide Children's Hospital, will describe the data via a brief oral presentation of the abstract titled "A Phase IIb Placebo-Controlled Study of the Exon-Skipping Drug Eteplirsen in Subjects with Duchenne Muscular Dystrophy" during the AAN Emerging Science Session (abstract #004 at 5:54 pm CDT), followed by a more detailed poster presentation (6:30 to 7:00 pm CDT).

SMT C1100 For Duchenne Muscular Dystrophy Moves To Human Testing - MarketWatch

SMT C1100 For Duchenne Muscular Dystrophy Moves To Human Testing - MarketWatch: The Muscular Dystrophy Association announced today that SMT C1100, an experimental drug for Duchenne muscular dystrophy (DMD), has received approval from regulatory agencies in the United Kingdom to move from laboratory to human testing, starting with healthy human volunteers.


The drug is designed to increase production of a muscle protein utrophin that can potentially compensate for the one that's missing in DMD.

Webinar: Results from DuchenneConnect Study - PPMD Community

Webinar: Results from DuchenneConnect Study - PPMD Community: Results from DuchenneConnect Study
DuchenneConnect�and�Parent Project Muscular Dystrophy�are proud to present the next in our ongoing�Direct Access Webinar Series�on�Wednesday, April 25 at 1pm eastern.
�
Topic:�Results from DuchenneConnect Study
When:�Wednesday, April 25 at 1pm eastern

University of Nevada School of Medicine research discovery and singular focus of April conference signal new hope for patients with rare diseases

Myomatrix 2012 conference to take place from April 22-24: The nation's leading scientists and clinicians exploring treatment breakthroughs for congenital muscular dystrophy will convene April 22-24 on the campus of the University of Nevada, Reno, where research by Dean Burkin has led to a potential therapy.

MDA Funds Development of Utrophin 'Magnet' for DMD/BMD | Quest Magazine Online

MDA Funds Development of Utrophin 'Magnet' for DMD/BMD | Quest Magazine Online: MDA Funds Development of Utrophin 'Magnet' for DMD/BMD
MDA's $1 million grant will help fund the development of TVN-102, which attracts the muscle protein utrophin to the muscle-fiber membrane.

Article Highlights:

• MDA funding will allow Tivorsan Pharmaceuticals to complete laboratory testing of TVN-102 and, if all goes well, to request permission from the FDA to begin human testing of the drug in 2012 or 2013.
• TVN-102 (biglycan) acts like a magnet, attracting utrophin to the muscle-fiber membrane. Animal studies suggest that utrophin at the muscle-fiber membrane can partially compensate for a lack of the protein dystrophin at the membrane, helping to stabilize the membrane.

AVI Summary Announcement - PPMD Community

AVI Summary Announcement - PPMD Community: Results
Results have demonstrated the following:
�
The group receiving Eteplirsen 30 mg/kg/week over 24 weeks resulted in a 22.5% increase in dystrophin on muscle biopsy, exceeding the company’s expectations. There was no increase in dystrophin in the placebo group.
The group receiving Eteplirsen 50mg/kg/week did not show an increase in dystrophin at 12 weeks, despite the higher dose.
The dystrophin produced appears to be new, novel dystrophin producing muscle fibers of normal length and consistency. The dystrophin appears to be diffusely distributed throughout the muscle biopsy.
There was no change in the clinical outcomes (6 minute walk test, Gower’s maneuver, 10 meter run) in the treatment group at 12 or 24 weeks.
Doses were well tolerated and there were no serious side effects to the drug at either doses.

Take Action: Ask Your Representative to Co-Sponsor FAST Act - PPMD Community

Take Action: Ask Your Representative to Co-Sponsor FAST Act - PPMD Community: We are coming to you today to ask that you devote your attention and energy towards legislation that seeks to expedite the process FDA uses to review drug applications. PPMD has officially endorsed�HR. 4132, the Faster Access to Specialized Treatments (FAST) Act, bipartisan legislation that was recently introduced in the House of Representatives.

How you can take action
The FAST Act is consistent with the FDA proposal the PPMD board adopted earlier this year. As such, we are requesting that you – our advocates – contact your members of Congress and urge them to co-sponsor the FAST Act.

We have prepared an email template for you to send to Representatives�and a phone script for additional follow-up.

Parent Project Muscular Dystrophy Endorses FAST Act Legislation to Expedite FDA Review... -- HACKENSACK, N.J., March 29, 2012 /PRNewswire-USNewswire/ --

Parent Project Muscular Dystrophy Endorses FAST Act Legislation to Expedite FDA Review... -- HACKENSACK, N.J., March 29, 2012 /PRNewswire-USNewswire/ --: HACKENSACK, N.J., March 29, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) – the leading advocacy organization fighting to end Duchenne muscular dystrophy – has endorsed legislation that would shorten the amount of time the Food and Drug Administration (FDA) takes to review candidate therapies for life-threatening conditions like Duchenne that lack other treatment options.

"The FAST Act is commonsense legislation that would leverage the Fast Track and Accelerated Review options at FDA's disposal. It would allow sponsors of candidate drugs to petition FDA to designate their product as being eligible for Fast Track review. It would also allow for approval of therapies on the condition of surrogate endpoints or intermediate markers used to gauge the trajectory of a disease," Furlong added.

Surrogate endpoints are a particularly important issue to the Duchenne community given the course of the disease and the need to more quickly determine whether or not a therapy is having its intended effect.

The legislation also seeks to more appropriately balance the benefits of a therapy with the potential risks. It would revoke accelerated approval if a sponsor fails to adequately study and monitor the therapy in patients or if further studies fail to verify clinical benefit.

"With nearly 20 potential therapies in clinical testing, it is a very exciting time for the Duchenne community, but a time in which we must make sure the FDA is equipped with the tools and laws it needs to evaluate these therapies as quickly as possible to save our sons," Furlong said.

"I commend Congressmen Stearns and Towns for introducing this legislation, and I urge other members to cosponsor it today so it can become law this year," she added.

Prosensa’s Clinical Development Plan Update – March 2012

In parallel to the on-going development of the lead compound PRO051/GSK2402968, which is being developed by GSK with an active involvement of Prosensa, there are five more compounds for Duchenne in Prosensa’s pipeline. Below you can find an update about the current state of development of these compounds.


http://community.parentprojectmd.org/profiles/blogs/prosensa-clinical-development-plan-update-march-2012?commentId=1187424%3AComment%3A150116&xg_source=msg_com_blogpost

Steroids: A Certain Can of Worms (A Complicated State of Affairs) - PPMD Community

Steroids: A Certain Can of Worms (A Complicated State of Affairs) - PPMD Community: Steroids are a complicated story and have clearly changed the natural history of Duchenne. We know they work for some, for most in fact, but not all. We know they increase strength and prolong ambulation, and statistics back this up. Predicting ambulation is prolonged on average for 2 years. But this is not the end of the story. Genetic modifiers have been identified that are thought to dramatically push out this timeline. Individuals with specific genetic modifiers continue walking into their mid- to late teens, often called outliers.

ImagingDMD MRI Study (DuchenneConnect Webinar Series) - PPMD Community

ImagingDMD MRI Study (DuchenneConnect Webinar Series) - PPMD Community: Dr. Krista Vandenborne and Claudia Senesac, PT, PhD, PCS, both from the University of Florida, discuss the ImagingDMD Study. This study focuses on developing Magnetic Resonance Imaging (MRI) as a tool to monitor disease progression in Duchenne and to serve as an outcome measure for clinical trials.

The aim of the study is to determine whether noninvasive MRI outcome measures can replace muscle biopsies in evaluating the effectiveness of new treatments in future clinical trials. A second goal of this study is to characterize the progressive involvement of the lower extremity muscles to help guide the design of future clinical trials.

Important Webinar: AVI Clinical Trial Update - PPMD Community

Important Webinar: AVI Clinical Trial Update - PPMD Community: Parent Project Muscular Dystrophy presents an AVI webinar on Wednesday, March 21 at 1pm eastern. The webinar will cover a pre-data update on AVI BioPharma's Phase IIb study evaluating eteplirsen for the treatment of Duchenne patients with mutations amenable to exon-51 skipping, and will be lead by Chris Garabedian, the CEO of AVI BioPharma.

New three-step process for DMD newborn screening

New three-step process for DMD newborn screening: Investigators at Nationwide Children's Hospital, working with the DNA Sequencing Core Facility at the University of Utah, have developed an approach to newborn screening (NBS) for the life-threatening genetic disorder, Duchenne muscular dystrophy (DMD) and potentially other muscular dystrophies.

Action Duchenne’s Seeds of Hope Appeal set to blossom for the third successive year - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne’s Seeds of Hope Appeal set to blossom for the third successive year - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "National sunflower campaign encourages children to learn about the environment and growing plants while raising awareness and funds for charity Action Duchenne

ARTICLE POSTED: 14 MARCH 2012 PERMANENT LINK
Action Duchenne is launching its national campaign, the Seeds of Hope Appeal for the third year running. Seeds of Hope is aimed at children between the ages of 3 and 11 years old, with the objective to raise awareness of the life limiting condition Duchenne Muscular Dystrophy. The appeal, which has been very successful in the last two years with over 3,000 participants last year, will raise funds for the charity, which campaigns to find a cure and viable treatments for Duchenne Muscular Dystrophy. "

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Our View - PPMD Community

Our View - PPMD Community: "So many times we view the world from the dark side, the part where we worry our sons (and daughters) may never see x or y, may never have this or that opportunity. I recall Kevin Smith standing up and telling us how he promised Zach there would be no limits on what he wanted to do or see, that if he wanted to see the top of a mountain, Kevin would put him on his back and show him the view. I think any of us would do that for our child or for our friend."
This video expresses loads of things, but one thing in particular, is that the view is still beautiful, even if you are carried.
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Volunteers needed to Dine Out for Duchenne! - PPMD Community

Volunteers needed to Dine Out for Duchenne! - PPMD Community: "Do you love to go out to eat? Do you want to help fundraise, but don't have the time? Then this is the event for you!! You can participate whenever you visit a new restaurant without doing extra work! It's fast and easy!!"

Dine Out for Duchenne is a week-long, online event where people can purchase gift certificates donated by restaurants throughout the United States. Supporters can click on an image map of states to find the listings, by city, in any state – for their own dining, as a gift, for when they travel, etc. Each restaurant will have a listing (multiple listings if more than one location) that includes a description of the restaurant, a link to their website, and the amount of the certificate. Press releases will be sent out to national and local media, focusing on the restaurants in local areas. The week before the event will be a preview week where people can look at the available certificates and target their purchases.

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Rescue of severely affected Dystrophin/Utrophin deficient mice through scAAV-U7snRNA mediated exon skipping

Rescue of severely affected Dystrophin/Utrophin deficient mice through scAAV-U7snRNA mediated exon skipping: "In this study, we report for the first time the efficiency of the AAV-mediated exon-skipping approach in the utrophin/dystrophin double-knockout mouse (dKO) which is a very severe and progressive mouse model of DMD. Following a single intravenous injection of scAAV9-U7ex23 in dKO mice, near-normal levels of dystrophin expression were restored in all muscles examined, including the heart."

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Reminder: Register Today for 2012 West Coast Connect Meeting - PPMD Community

Reminder: Register Today for 2012 West Coast Connect Meeting - PPMD Community: "By now we hope you have heard that Parent Project Muscular Dystrophy will be hosting our first West Coast Connect Meeting, April 13-15, 2012 in San Diego, CA at the San Diego Marriott Mission Valley. This will be a great opportunity to get the latest research updates on a variety of different therapies in the pipeline, as well as direct access to the people moving these projects along. We are very excited about the comprehensive agenda that we have put together, and the amazing assembly of speakers."

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Motion in Scottish Parliament for Action Duchenne - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Motion in Scottish Parliament for Action Duchenne - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Action Duchenne was highlighted in Holyrood earlier this month thanks to the support of MSP Jim Eadie and grandparent John Miller.

The motion presented in Holyrood was focused on the upcoming Rock 'n' Roll half marathon that is taking place in Edinburgh on 15th April. The motion gained great support by: Stuart McMillan, Jackie Baillie, Angus MacDonald, Dennis Robertson, David Torrance, Kenneth Gibson, John Lamont, Richard Lyle, Nanette Milne, Bill Kidd, Humza Yousaf, Joan McAlpine, Adam Ingram, Mike MacKenzie, Christina McKelvie, Jackson Carlaw, Marco Biagi, Margaret Burgess, Kevin Stewart, James Dornan, Jean Urquhart, Elaine Smith, Maureen Watt, Dave Thompson, Gil Paterson, Annabelle Ewing, Jamie Hepburn.

MSP Jim Eadie and those supporting the event and work of Action Duchenne is greatly appreciated. Good luck and thank you to everyone taking part on the day.

If you, or someone you know, would like to get involved with the Rock 'n' Roll half marathon in April then please get in touch."

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TAKE ACTION! 2012 Advocacy Conference Action Alert

Parent Project Muscular Dystrophy: Email - TAKE ACTION! 2012 Advocacy Conference Action Alert: "We need your help in supporting PPMD's 2012 Advocacy Agenda.

Whether or not you were able to join us in Washington, DC for our 2012 Advocacy Conference, now is the time to make your voice heard. As always, we aim to preserve critical funding for Duchenne research and education programs. "

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PPMD Announces Board Approved FDA Policy - PPMD Community

PPMD Announces Board Approved FDA Policy - PPMD Community: "At this time, Congress is working on writing a critical FDA related bill called the Prescription Drug User Fee Act (PDUFA), and PPMD is working with other rare disease advocates on provisions to benefit our sons. Earlier this year, the PPMD Board of Directors adopted a policy position that, among other things, seeks to ensure the potential benefits of a therapy are weighted appropriately against possible risks.

PPMD is working now to develop a specific legislative provision or provisions that would advance these core principles, and will be committed to working this issue as Congress writes its FDA bill over the coming months.

Please take a moment to read this policy. We cannot stress enough how important it is for your family, for every Duchenne family and organization to understand this topic, as it will most definitely impact how quickly treatments are available to our sons."

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Parent Project Muscular Dystrophy Awards $500,000 to Tivorsan Pharmaceuticals -- HACKENSACK, N.J., Feb. 21, 2012 /PRNewswire-USNewswire/ --

Parent Project Muscular Dystrophy Awards $500,000 to Tivorsan Pharmaceuticals -- HACKENSACK, N.J., Feb. 21, 2012 /PRNewswire-USNewswire/ --: "Dr. Justin Fallon of Brown University, the founding scientist of Tivorsan, was the recipient of PPMD's first End Duchenne Grant in 2008. Building on that work, the Tivorsan team seeks to use recombinant human biglycan (rh-BGN) to increase utrophin at the muscle cell membrane, resulting in reduced muscle damage and improved function."

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Ataluren update

Ataluren update - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "PTC Therapeutics is actively involved in preparations for initiating an open-label study for patients with nonsense mutation Duchenne/Becker muscular dystrophy previously treated with ataluren at sites outside the U.S. "

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JumpStart Invests $250,000 in Milo Biotechnology -- CLEVELAND, Feb. 14, 2012 /PRNewswire/ --

JumpStart Invests $250,000 in Milo Biotechnology -- CLEVELAND, Feb. 14, 2012 /PRNewswire/ --: "CLEVELAND, Feb. 14, 2012 /PRNewswire/ -- Milo Biotechnology will receive $250,000 from JumpStart Inc., a nonprofit investing intensive business assistance and some capital into early stage Northeast Ohio-based tech companies. A clinical stage startup developing a therapy to increase muscle strength and improve the quality of life of muscular dystrophy patients, Milo is the 61st company in JumpStart's portfolio. "There are limited treatment options for muscular dystrophy and approved therapies are inadequate or can cause significant side effects," says JumpStart's Mike Lang. "Since most muscular dystrophy patients are children, the medical community is interested in a safer and more targeted therapy.""

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SUMMIT OUTLINES CLINICAL TRIAL PLANS FOR ITS DUCHENNE MUSCULAR DYSTROPHY PROGRAMME - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

SUMMIT OUTLINES CLINICAL TRIAL PLANS FOR ITS DUCHENNE MUSCULAR DYSTROPHY PROGRAMME

"Clinical Trial Application expected to be submitted in Q1 2012
• Headline results from Phase I study anticipated in Q3 2012
Oxford, UK, 14 February 2012, Summit (AIM: SUMM), a UK drug discovery company, today outlines
its clinical trial plans for SMT C1100, a potential first-in-class disease modifying drug for the treatment
of the fatal rare disease Duchenne Muscular Dystrophy (‘DMD’). "

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Making a mark in Northern Ireland - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Making a mark in Northern Ireland - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Action Duchenne will again be making a mark in Northern Ireland this year thanks to the hard work and support of parents Andrew and Yvette McClean.

For the first time in the history of Action Duchenne the Belfast City Marathon, being held on Monday 7th May 2012, will see swarm of our charity tops take to the streets with 31 people taking part helping to raise awareness and funds for Action Duchenne. "

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Action Duchenne joins healthcare professionals in opposing Health and Social Care Bill - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne joins healthcare professionals in opposing Health and Social Care Bill - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Action Duchenne has announced its opposition to the Health and Social Care Bill due to be debated by again by the House of Lords from 8th February.

The Health and Social Care Bill represents the biggest change to the NHS since its inception, at a time when public health services are facing additional financial challenges.
"

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Action Duchenne patron honoured with CBE - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne patron honoured with CBE - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Helena Bonham Carter, who became Action Duchenne's first patron in 2008, has been awarded a CBE in the New Years Honours announced today. Everyone connected with Action Duchenne would like to congratulate Helena for her fabulous achievement following a year in which she was nominated for an Oscar in the award winning Kings Speech. We looking forward to Helena's continuing support for our work in 2012. "

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