Fling for Logan'forty step fling' challenge

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

2012-01-03T08:58:00.000Z

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. "

'via Blog this'

Parent Project Muscular Dystrophy: Leading the fight to end Duchenne

2011-12-20T09:08:00.000Z

Parent Project Muscular Dystrophy: Leading the fight to end Duchenne: "PPMD is happy to be supporting Summit PLC with its utrophin upregulation drug candidate SMT C1100. The funding provided will enable Summit to manufacture a new formulation of SMT C1100 and conduct a Phase I clinical trial in healthy volunteers."

'via Blog this'

Help Santa deliver his toys and support Action Duchenne with new Christmassy iPhone & iPad App from Rantmedia - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-12-15T13:56:00.000Z

Help Santa deliver his toys and support Action Duchenne with new Christmassy iPhone & iPad App from Rantmedia - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Download Santa’s Sleigh Ride, enjoy the fun and 15% of proceeds go to Action Duchenne from http://itunes.apple.com/gb/app/santas-sleigh-ride/id477668167

ARTICLE POSTED: 15 DECEMBER 2011 PERMANENT LINK
RantMedia has designed a fabulous new iPhone & iPad app called Santa’s Sleigh Ride where you get Santa and his reindeer to jump over toys, decorations and other festive things. The game is easy to play, yet highly addictive with increasingly fast and furious levels. "

'via Blog this'

World Muscle Society 2011 Report - PPMD Community

2011-12-06T10:03:00.000Z

World Muscle Society 2011 Report - PPMD Community: "At the recent World Muscle Society meeting, which took place in Portugal, a great deal of new data on exon skipping was presented, both on the status of current human clinical trials and on the efforts of investigators to improve the efficiency of the technique by combining exon-skipping with gene and stem cell delivery. Below are a few highlights from the meeting with the caveat that it was impossible to cover every poster and talk. "

'via Blog this'

New gene therapy approach safe for patients with muscular dystrophy

2011-12-01T09:54:00.000Z

New gene therapy approach safe for patients with muscular dystrophy: "Researchers at the University of North Carolina at Chapel Hill have shown that it is safe to cut and paste together different viruses in an effort to create the ultimate vehicle for gene therapy. In a phase I clinical trial, the investigators found no side effects from using a "chimeric" virus to deliver replacement genes for an essential muscle protein in patients with muscular dystrophy."

'via Blog this'

Medicine thrown into crisis by stem cell ruling - Science, News - The Independent

2011-10-19T08:48:00.000+01:00

Medicine thrown into crisis by stem cell ruling - Science, News - The Independent: "British medical researchers have condemned a Europe-wide ban on the patenting of stem cell inventions derived from human embryos – setting back possible new treatments for a range of disorders, from heart disease and diabetes to blindness and Parkinson's."

'via Blog this'

AVI BioPharma - News Release

2011-10-19T08:46:00.001+01:00

AVI BioPharma - News Release: "AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced data presentations for eteplirsen (the non-proprietary name assigned to AVI-4658), the Company's investigational exon skipping therapy for the treatment of Duchenne muscular dystrophy, at the 16th International Congress of the World Muscle Society taking place Oct. 18-22, 2011 in Almancil, Algarve, Portugal."

'via Blog this'

Study in Need of Boys with Duchenne: MRI and Biomarkers for DMD (ImagingDMD)

2011-10-19T08:46:00.000+01:00

Study in Need of Boys with Duchenne: MRI and Biomarkers for DMD (ImagingDMD): "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. This study is funded by the NIH – NIAMS/NINDS. "

'via Blog this'

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

2011-10-11T15:49:00.000+01:00

Duchenne petition - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Action Duchenne member Muriel Sherlock has created a petition on the government's e-petition site, calling for more funding for vital Duchenne research.

With almost 500 signatures already, it's really important that we all share it with our contacts to get as many signatures as possible.

As Muriel says, 'The British government need to give some of our taxpayers money to find a cure for something that should have been cured years ago'. "

'via Blog this'

First NIH-funded personalized drug development center in US will focus on muscle disease

2011-10-07T09:00:00.000+01:00

First NIH-funded personalized drug development center in US will focus on muscle disease: "The first Center of Research Translation (CORT), focused on personalized drug development for Duchenne muscular dystrophy (DMD), has been created through a $7.9 million grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health. The 5-year grant was awarded to a consortium of academic laboratories at Children's National Medical Center in Washington, DC, University of Pittsburgh in Pittsburgh, PA, and Carolinas Medical Center in Charlotte, NC."

'via Blog this'

Champion dancer cutting hair for charity

2011-10-03T09:14:00.000+01:00

On 30th October 2011 11 year old Champion Dancer Chloe McClean from Edinburgh is raising the profile and funds by supporting two charities.

Chloe has been growing her hair for a while so that she could have it cut and donate it to www.littleprincesses.org.uk which is a Charity that makes wigs for kids with Cancer.

She also thought it would be a good idea to do this as a sponsored event and raise money for Action Duchenne at the same time in support of our son Logan Mitchelson (www.flingforlogan.com)"

'via Blog this'

Communication breakdown between nerves and muscles in SMA mouse model

2011-09-28T08:42:00.000+01:00

Communication breakdown between nerves and muscles in SMA mouse model: "Researchers at the University of Missouri have identified a communication breakdown between nerves and muscles in mice that may provide new insight into the debilitating and fatal human disease known as spinal muscular atrophy (SMA)."

'via Blog this'

Prosensa Advances Three Exon Skipping Candidates for Duchenne Muscular Dystrophy into the Next Development Stage - Prosensa to receive up to £27M in development and milestone payments from GSK - Prosensa

2011-09-13T16:24:00.000+01:00

Prosensa Advances Three Exon Skipping Candidates for Duchenne Muscular Dystrophy into the Next Development Stage - Prosensa to receive up to £27M in development and milestone payments from GSK - Prosensa: "Prosensa, the Dutch biopharmaceutical company focusing on rare diseases with an unmet medical need, announced today that they have agreed with GlaxoSmithKline (GSK) to advance three further exon skipping compounds (PRO044, PRO045 and PRO053) into the next development stage under their ongoing collaboration relationship in Duchenne Muscular Dystrophy (DMD)."

'via Blog this'

Vote now for Include Duchenne in the National Lottery Awards - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-09-02T12:37:00.000+01:00

Vote now for Include Duchenne in the National Lottery Awards - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "VOTE NOW FOR INCLUDE DUCHENNE IN THE NATIONAL LOTTERY AWARDS
Second and final round of voting to decide the winner in the National Lottery Awards starts now"

'via Blog this'

AVI BioPharma - News Release

2011-08-16T08:18:00.000+01:00

AVI BioPharma - News Release: "AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced that it has initiated dosing in a Phase 2 study of eteplirsen, the Company's lead exon-skipping therapeutic candidate for the treatment of Duchenne muscular dystrophy (DMD)"

Heart Failure Drugs Preserve Muscle in Muscular Dystrophy

2011-08-03T20:53:00.000+01:00

Heart Failure Drugs Preserve Muscle in Muscular Dystrophy: "COLUMBUS, Ohio – Two common drugs used to treat heart failure might also improve heart and muscle function in Duchenne muscular dystrophy (DMD), according to a new study. "

Researchers discover biological mechanism behind sarcopenia

2011-08-03T20:52:00.000+01:00

Researchers discover biological mechanism behind sarcopenia: "Researchers at Columbia University Medical Center have discovered the biological mechanism behind age-related loss of muscle strength and identified a drug that may help reverse this process."

BBC News - Molecular scalpel hope for Duchenne muscular dystrophy

2011-07-25T12:19:00.000+01:00

BBC News - Molecular scalpel hope for Duchenne muscular dystrophy: "A 'molecular scalpel' shows promise in patients with a deadly muscle wasting condition, according to researchers."

AVI BioPharma - News Release

2011-07-25T12:17:00.000+01:00

AVI BioPharma - News Release: "The Lancet Publishes Clinical Trial Data That Demonstrate Statistically Significant and Dose-Dependent Expression of Dystrophin in Duchenne Muscular Dystrophy Patients Treated With AVI BioPharma's Eteplirsen"

Coach to Cure MD Set for September 24 Games - AFCA.com—Official Site of the American Football Coaches Association

2011-07-22T08:47:00.000+01:00

Coach to Cure MD Set for September 24 Games - AFCA.com—Official Site of the American Football Coaches Association: "For the fourth year in a row, college football coaches nationwide will join together in support of the Coach to Cure MD program, which will be held during games of Saturday, September 24, 2011. The rapidly growing annual effort has raised nearly a million dollars to battle Duchenne muscular dystrophy, and should easily top the million-dollar plateau in 2011."

Parent Project Muscular Dystrophy: Education Matters

2011-06-16T10:01:00.000+01:00

Parent Project Muscular Dystrophy: Education Matters: "Education Matters Guides
To better assist parents and teachers in providing a fulfilling education for the student with Duchenne, PPMD has created the following helpful guides:"

- A Parent's Guide

- A Teacher's Guide

- Adaptive PE

- Learning & Behavior in Duchenne

Make sure that your school has these.

Biglycan Treatment Shows Promise in Muscular Dystrophy Mouse Model

2011-06-15T08:36:00.000+01:00

Biglycan Treatment Shows Promise in Muscular Dystrophy Mouse Model: "Biglycan Treatment Shows Promise in Muscular Dystrophy Mouse Model

Treatment with the protein biglycan reduces muscle damage in a mouse model of Duchenne and Becker muscular dystrophies (DBMD), according to new research supported, in part, by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). Dystrophin deficient (mdx) mice given systemic injections of purified biglycan were less susceptible to the loss of muscle strength that characterizes the disease. The study was reported in the Proceedings of the National Academy of Sciences"

Parent Project Muscular Dystrophy: Home

2011-06-15T08:35:00.000+01:00

Parent Project Muscular Dystrophy: Home: "Dr. Brenda Wong and her colleagues at Cincinnati Children's Hospital Medical Center in Ohio are actively recruiting boys with Duchenne for their IGF-1 study: Safety and Efficacy Study of IGF-I in Duchenne."

Parent Project Muscular Dystrophy: Home

2011-06-08T08:33:00.000+01:00

Parent Project Muscular Dystrophy: Home: "PPMD and DuchenneConnect are pleased to announce that the Clinic Services Resource is now available for patients and families of those affected by Duchenne and Becker muscular dystrophy. All those who receive care in the United States are encouraged to join this dynamic new expansion of the DuchenneConnect Registry."

AVI BioPharma Live Podcast 6/20/11

2011-06-07T11:08:00.000+01:00

AVI BioPharma Live Podcast 6/20/11: "Chris Garabedian, CEO of AVI BioPharma, will participate in a live podcast hosted by PPMD on June 20, 2011 at 1:00 pm ET. Chris will review AVI’s development of eteplirsen, the company’s Phase 2 exon 51 skipping therapeutic candidate to restore dystrophin expression and treat DMD. Chris will also respond to questions submitted by email on or before June 16, 2011. Questions can be submitted by emailing info@parentprojectmd.org with the subject line: AVI Webinar. "

‘Include Duchenne’ project Decipha gets to finals of the National Lottery Awards 2011 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-05-31T10:08:00.000+01:00

‘Include Duchenne’ project Decipha gets to finals of the National Lottery Awards 2011 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Voting for the semi-finals starts at 9am on Tuesday, 31st May and ends at midday on Monday, 20th June. We need your vote – you can either call the 0844 836 9683 or vote online at"

Parent Project Muscular Dystrophy: Home

2011-05-19T08:43:00.000+01:00

Parent Project Muscular Dystrophy: Home: "The House deadline for Fiscal Year (FY) 2012 Labor-Health & Human Services (L-HHS) appropriations requests is Friday, May 20! As such, we need all Advocates to encourage their Representatives to support our FY 2012 funding priorities. Please take a few moments to contact your House member’s office and ask that he/she sign-on to our FY 2012 Duchenne muscular dystrophy appropriations letter and submit our FY 2012 report language."

Now Recruiting: MRI and Biomarkers for Duchenne Muscular Dystrophy Study (ImagingDMD)

2011-05-10T16:37:00.000+01:00

Now Recruiting: MRI and Biomarkers for Duchenne Muscular Dystrophy Study (ImagingDMD): "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. This study is funded by the NIH – NIAMS/NINDS. "

Santhera Presents Data from 2-Year Open-Label Intervention Study with Catena® in Duchenne Muscular Dystrophy at the MYOLOGY 2011 Conference | Reuters

2011-05-10T08:29:00.000+01:00

Santhera Presents Data from 2-Year Open-Label Intervention Study with Catena® in Duchenne Muscular Dystrophy at the MYOLOGY 2011 Conference | Reuters: "Santhera Pharmaceuticals (SIX: SANN) announced today that it will present first analyses of the 2-year open-label study (DELPHI-E) evaluating Catena® for the treatment of Duchenne Muscular Dystrophy. The findings indicate that Catena® can slow the decline in respiratory function associated with patients as they age. Data will be presented on May 9, 2011 in Lille, France at the 4th International Congress of Myology [1].
"

Daily treatment with SMT C1100 dramatically reduces muscular dystrophy symptoms in mdx mouse - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-05-05T11:06:00.000+01:00

Daily treatment with SMT C1100 dramatically reduces muscular dystrophy symptoms in mdx mouse - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Summit plc based in Abingdon, UK, together with the Davies group (University of Oxford) and collaborators have reported exciting results from its latest studies of its utrophin upregulator drug SMT C1100. It has developed a small molecule based drug that can be taken orally once a day which could treat all patients with muscular dystrophy regardless of their specific gene defect. The studies took place using the mdx mouse, a recognised model for Duchenne Muscular Dystrophy study and in human tissues in the lab. The results showed increased utrophin in muscles and that this helped increase the amount of exercise achievable and therefore overall strength. These significant results demonstrate the potential importance of SMT C1100 and should pave the way for the next human trials of the drug."

Sildenafil/Taladafil Study at Cedars-Sinai Medical Center IRB-Approved

2011-05-05T07:57:00.000+01:00

Sildenafil/Taladafil Study at Cedars-Sinai Medical Center IRB-Approved: "The sildenafil/taladafil study at Cedars-Sinai Medical Center is now IRB-approved and will begin recruiting patients in June."

Update: Partial Clinical Hold Lifted by FDA for GSK2402968 (Exon 51 Skip)

2011-04-26T10:24:00.000+01:00

Update: Partial Clinical Hold Lifted by FDA for GSK2402968 (Exon 51 Skip): "GlaxoSmithKline (GSK) has provided an update regarding the clinical development program for GSK2402968 (exon 51 skip) for DMD: The US Food and Drug Administration has lifted the partial clinical hold on the Investigational New Drug Application for GSK2402968. This means GSK can now proceed with longer term clinical studies of GSK2402968 in DMD patients in the US."

ACE-031 statement from Acceleron and Shire - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-04-26T08:21:00.000+01:00

ACE-031 statement from Acceleron and Shire - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "ACE-031 STATEMENT FROM ACCELERON AND SHIRE
Clinical trial suspended; intention is to start a new study after discussions with regulatory agencies"

Tweaking gene in satellite cells may increase lifespan of people with muscular dystrophy

2011-04-19T08:38:00.000+01:00

Tweaking gene in satellite cells may increase lifespan of people with muscular dystrophy: "Tweaking gene in satellite cells may increase lifespan of people with muscular dystrophy"

Teen With Duchenne Muscular Dystrophy Keeps on Achieving - YNN, Your News Now

2011-04-15T08:40:00.000+01:00

Teen With Duchenne Muscular Dystrophy Keeps on Achieving - YNN, Your News Now: "Teen With Duchenne Muscular Dystrophy Keeps on Achieving"

Parent Project Muscular Dystrophy: GSK 2402968 (PRO-051)

2011-04-12T15:38:00.000+01:00

Parent Project Muscular Dystrophy: GSK 2402968 (PRO-051): "the forty-eight week follow-up data from the phase I/IIa extension study (PRO051-02) will be presented at an oral session on April 13, 2011 during the 63rd annual meeting of the American Academy of Neurology."

Run for Scotland - DeanWidd - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-04-12T11:34:00.000+01:00

Run for Scotland - DeanWidd - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy: "If you are a runner rather than a walker then why not get involved with the 5k fun run around Holyrood Park Edinburgh on Sunday 5th June.

Starting at Holyrood Park at 9.30am runners of all abilities are welcome to get involved and help raise awareness and funds for Action Duchenne. All runners will receive a medal , completion certificate and a goody bag."

AVI BioPharma Receives Approval of Nonproprietary Name Eteplirsen for Lead Duchenne Muscular Dystrophy Therapeutic Candidate, AVI-4658 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-04-12T11:32:00.000+01:00

AVI BioPharma Receives Approval of Nonproprietary Name Eteplirsen for Lead Duchenne Muscular Dystrophy Therapeutic Candidate, AVI-4658 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "AVI BioPharma (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced that the United States Adopted Names (USAN) Council has approved the nonproprietary name eteplirsen for AVI-4658, the Company's exon-skipping therapy for the treatment of Duchenne muscular dystrophy (DMD), a genetic muscle wasting disease caused by the absence of functional dystrophin. In addition, the World Health Organization (WHO) has approved eteplirsen as the International Nonproprietary Name (INN) for AVI-4658."

Golden Mile 1,2 or 3 mile walk - DeanWidd - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-04-12T11:03:00.000+01:00

Golden Mile 1,2 or 3 mile walk - DeanWidd - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy: "Starting at 11:30am on Sunday 5th June 2011, the Golden Mile Walks are primarily aimed at all who wish to participate in what will be a tremendous fun day out. Whether you wish to walk or stroll 1,2, or 3 miles the choice is yours.Anybody can take part and no one is excluded"

Parent Project Muscular Dystrophy: Home

2011-04-12T08:39:00.000+01:00

Parent Project Muscular Dystrophy: Home: "Once again, TRND is accepting proposals, due April 26, 2011. This program is part of a congressionally mandated program to encourage and speed the development of new drugs for rare and neglected diseases. This unique program creates a drug development pipeline within the NIH and is specifically intended to stimulate research collaborations with academic scientists, non-profit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses."

£2.5m research grant for Duchenne Muscular Dystrophy awarded to Oxford researchers - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-04-08T13:42:00.000+01:00

£2.5m research grant for Duchenne Muscular Dystrophy awarded to Oxford researchers - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "£2.5M RESEARCH GRANT FOR DUCHENNE MUSCULAR DYSTROPHY AWARDED TO OXFORD RESEARCHERS
Funding for pioneering research into gene therapy by Action Duchenne has led to further funds for research into this life limiting disease"

Action Duchenne welcomes backtrack on NHS plans - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-04-06T10:16:00.000+01:00

Action Duchenne welcomes backtrack on NHS plans - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "ACTION DUCHENNE WELCOMES BACKTRACK ON NHS PLANS
Charity states that government plans that threaten the most vulnerable must now be re-examined, young men to lobby parliament for access to treatment"

Phrixus Pharmaceuticals, Inc. Announces $890,000 in NIH Funding for Its Programs in Duchenne Muscular Dystrophy and Heart Failure

2011-04-05T15:25:00.000+01:00

Phrixus Pharmaceuticals, Inc. Announces $890,000 in NIH Funding for Its Programs in Duchenne Muscular Dystrophy and Heart Failure: "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 a total of $890,000 in awards from the National Institutes of Health (NIH). Funding is in the form of one SBIR Phase 1 award titled 'Effects of P-188 on Respiratory Function and Diaphragm Degeneration in the mdx Mouse' and one STTR Phase 1 award titled 'Poloxamer 188 Mechanism of Action in Ischemic Heart Failure.' The latter award is in collaboration with Dr. Joseph M. Metzger, Chair of Integrative Biology and Physiology at the University of Minnesota."

Cedars-Sinai receives $750,000 grant from Parent Project Muscular Dystrophy for sildenafil/taladafil study

2011-04-05T11:56:00.000+01:00

Cedars-Sinai receives $750,000 grant from Parent Project Muscular Dystrophy for sildenafil/taladafil study: "Cedars-Sinai receives $750,000 grant from Parent Project Muscular Dystrophy for sildenafil/taladafil study"

Parent Project Muscular Dystrophy: Cialis® (Tadalafil)

2011-04-05T08:29:00.000+01:00

Parent Project Muscular Dystrophy: Cialis® (Tadalafil): "In Duchenne (and some cases of Becker) the lack of dystrophin causes, among other things, a decrease in nitric oxide, which normally stimulates cGMP and holds blood vessels open during muscle contraction. Without adequate NO levels, it is thought that the muscles of those with Duchenne and Becker may experience “ischemia,” or damage due to a lack of oxygen. By blocking PDE-5, tadalafil may be able to compensate for the loss of NO at the muscle membrane by upregulating cGMP directly and protecting the muscles of those with Duchenne/Becker during exercise."

Google Reader (10)

2011-04-01T08:57:00.000+01:00

Google Reader (10): "Mitch Sevier, a talented photographer and father of a son with Duchenne, has put together some incredible images that we feel truly capture what Duchenne “looks” like. These photographs illustrate Duchenne in a way PPMD has never experienced."

PPMD - Duchenne app now downloading

2011-03-24T14:51:00.000Z

Parent Project Muscular Dystrophy: Home: "Download PPMD’s New Duchenne App
PPMD is excited to announce the release of our new, free mobile applications designed specifically for the iPhone, iPad, and Androids. PPMD's new app will put emergency care information, critical research news, and advocacy alerts at your fingertips, instantaneously"

Results of Prosensa’s Extended Phase I/II Exon-skipping Trial in Duchenne Muscular Dystrophy Published in the New England Journal of Medicine - Prosensa

2011-03-24T14:40:00.000Z

Results of Prosensa’s Extended Phase I/II Exon-skipping Trial in Duchenne Muscular Dystrophy Published in the New England Journal of Medicine - Prosensa: "Prosensa,, announces the publication of results from a phase I/II and 12-week extension study of its lead product, PRO051 (GSK2402968) for the treatment of Duchenne Muscular Dystrophy (DMD) in the New England Journal of Medicine (NEJM)[1]."

Parent Project Muscular Dystrophy: Mobile App

2011-03-23T13:50:00.000Z

Parent Project Muscular Dystrophy: Mobile App: "The next time you are meeting with your doctor and want to access information from PPMD's vast research or care archive, you can. The next time you have to take your child to the emergency room and are unsure what safety measures must be taken into consideration, the PPMD app is there. The next time you are in an IEP meeting and need to make sure you went through your checklist of discussion items, your go-to resource is available"

Transparent fish to make human biology clearer

2011-03-22T08:22:00.000Z

Transparent fish to make human biology clearer: "Zebrafish are genetically similar to humans and are good models for human biology and disease. Now, researchers at Children's Hospital Boston have created a zebrafish that is transparent throughout its life. The new fish allows scientists to directly view its internal organs, and observe processes like tumor metastasis and blood production after bone-marrow transplant in a living organism."

New insight into Duchenne muscular dystrophy

2011-03-22T08:20:00.000Z

New insight into Duchenne muscular dystrophy: "Armed with a zebrafish model of Duchenne muscular dystrophy (DMD) and a library of 1,200 chemicals already approved for human use, researchers at Children's Hospital Boston have identified a compound that reverses the loss of muscle structure and function associated with DMD, seemingly by compensating for the loss of a critical protein."

2012 Walt Disney World Marathon Weekend Registration is Open!

2011-03-17T09:17:00.001Z

Parent Project Muscular Dystrophy:: "2012 Walt Disney World Marathon Weekend Registration is Open!
Register today for a magical family weekend."

GSK Now Recruiting for Exon 51 Clinical Trial in Non-Ambulatory Boys

2011-03-17T09:17:00.000Z

Parent Project Muscular Dystrophy: Home: "GSK Now Recruiting for Exon 51 Clinical Trial in Non-Ambulatory Boys
This study will assess the pharmacokinetics, safety, and tolerability of GSK2402968 after a single administration, at escalating dose levels, in non-ambulatory boys with Duchenne who have a dystrophin gene mutation amenable to an exon 51 skip."

1 wee boy - 3 fractures

2011-03-08T11:53:00.000Z

We were at a swimming party on Saturday and Logan slipped whilst diving into the water. His foot hurt a bit but he carried on.

It swelled up overnight and we got it checked at hospital on Sunday - no real pain so just keep an eye on it.

It swelled up even more on Sunday night so Monday Donna took him for an x-ray - 3 fractures on his instep!

He now has his leg in plaster (looks a bit like licorice) but even that could not stop him smiling!

Love you Logan

AMT Receives Grant From Dutch Parents Organization for Duchenne Muscular Dystrophy Gene Therapy -- AMSTERDAM, March 7, 2011 /PRNewswire-FirstCall/ --

2011-03-08T08:29:00.000Z

AMT Receives Grant From Dutch Parents Organization for Duchenne Muscular Dystrophy Gene Therapy -- AMSTERDAM, March 7, 2011 /PRNewswire-FirstCall/ --: "AMT Receives Grant From Dutch Parents Organization for Duchenne Muscular Dystrophy Gene Therapy"

Funds for Action Duchenne as Xbox fans blast aliens with new game - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-03-03T08:23:00.000Z

Funds for Action Duchenne as Xbox fans blast aliens with new game - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Proceeds from a newly released game, Alien Super Mega Blaster for Microsoft Xbox 360 are being donated to the charity Action Duchenne by developer Jon Hearn. The classic space invaders game with a twist was developed by Jon in his spare time."

CARE-NMD launches new project website - visit now - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-03-01T10:26:00.000Z

CARE-NMD launches new project website - visit now - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "CARE-NMD launches new project website
Online resources made available to patients, families and clinicians across Europe on standards of care for DMD
A newly revised website has been launched as part of CARE-NMD, (www.care-nmd.eu) the three year project to implement best-practice standards of care for Duchenne muscular dystrophy across Europe."

Love Runs raise over £40k to help find a cure! - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-03-01T08:30:00.001Z

Love Runs raise over £40k to help find a cure! - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "The results have been announced, and we are eagerly gathering in the funds raised, and it is looking good! At the moment from entries and sponsorship it is looking like we will have raised over £40k to help find a cure for Duchenne."

Videos: Patients with Rare Diseases and Conditions > Duchenne muscular dystrophy: Charlie's Story

2011-03-01T08:30:00.000Z

Videos: Patients with Rare Diseases and Conditions > Duchenne muscular dystrophy: Charlie's Story: "Chuck and his son Charlie visited OOPD and told their story of living with Duchenne muscular dystrophy (DMD). Charlie was diagnosed when he was 18 months old, and now at 11 years old describes his life challenges and goals."

Google Reader (53)

2011-03-01T08:29:00.000Z

Google Reader (53): "Chuck and his son Charlie visited OOPD and told their story of living with Duchenne muscular dystrophy (DMD). Charlie was diagnosed when he was 18 months old, and now at 11 years old describes his life challenges and goals."

Parent Project Muscular Dystrophy: Race Calendar

2011-02-22T09:13:00.000Z

Parent Project Muscular Dystrophy: Race Calendar: "Come to run, come to cheer, or find a runner to sponsor. Even if you can't be there in person, you can still track the progress of the race or the fundraising efforts online!"

Run fo our sons - Disneyland 1/2 marathon

2011-02-22T09:06:00.000Z

Parent Project Muscular Dystrophy:: "NEW FOR 2011: If you ran this race with the Run For Our Sons team last year, receive FREE entry into the race this year!"

Quality of Standards of Care - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-02-22T08:32:00.000Z

Quality of Standards of Care - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "We will be inviting participants to attend focus group meetings which will take place in London and will last no longer than 2 hours (9.30am-11.30am). The focus group meetings will take place on Monday, 21st March and Friday, 1st April 2011 at the Royal Society of Medicine. The venue is situated at: 1 Wimpole Street, London, W1G 0AE."

Interleukin-10 reduces the pathology of mdx muscular dystrophy by deactivating M1 macrophages and modulating macrophage phenotype. - Duchennepedia - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-02-17T10:52:00.000Z

Interleukin-10 reduces the pathology of mdx muscular dystrophy by deactivating M1 macrophages and modulating macrophage phenotype. - Duchennepedia - Action Duchenne - Fighting for a cure for muscular dystrophy: "Together, the results show that IL-10 plays a significant regulatory role in muscular dystrophy that may be caused by reducing M1 macrophage activation and cytotoxicity, increasing M2c macrophage activation and modulating muscle differentiation."

We need YOUR help to further our Advocacy efforts in Washington, DC!

2011-02-17T08:41:00.000Z

Parent Project Muscular Dystrophy:: "How can YOU help? We need you to make a phone call and send an email!

ACTION 1. Phone Call: Please take a moment to CALL your Senators and Representatives and urge them to sign onto our multimember letter being circulated by Representative Matsui in the House and Senators Wicker in the Senate.

Wondering what to say when you call your Senators and Congressman?

Dont worry, we have provided you with a script: Senate Phone Script House Phone Script

To look up their phone number click here

ACTION 2. Send Action Alert NOW: Email your Representative and Senators-PPMD is requesting your support by clicking through our email action alert below. It takes two minutes!

This simple one step alert will automatically send our message to your Senators and Representative and will also allow you to forward the message to friends and family who may also want to support our Advocacy Agenda.

Click here to send an email to your members of Congress!

Thank you so much for taking the time to support these very important initiatives. "

GSK and Prosensa announce start of Phase III study of investigational Duchenne Muscular Dystrophy medication

2011-02-17T08:40:00.001Z

GSK and Prosensa announce start of Phase III study of investigational Duchenne Muscular Dystrophy medication: "GlaxoSmithKline (GSK) and Prosensa today announced that the first patient has commenced treatment in the Phase III clinical study investigating GSK2402968 (`968), in ambulant boys with Duchenne Muscular Dystrophy (DMD), who have a dystrophin gene mutation amenable to an exon 51 skip (up to 13% of boys with DMD). Commencement of this study confirms previously announced plans to progress this asset into Phase III.
This randomised, placebo controlled study will enrol 180 patients, from up to 18 countries, and is currently the most advanced ongoing study for this rare, severely debilitating, neuromuscular disease.
The study is designed to assess the efficacy and safety of GSK968 6mg/kg, once weekly, compared to placebo, for 48 weeks in ambulant boys over 5 years of age with DMD The primary efficacy endpoint is a measure of muscle function using the six minute walking distance test."

Lack of DOT1L enzyme could lead to some types of heart disease: Study

2011-02-17T08:40:00.000Z

Lack of DOT1L enzyme could lead to some types of heart disease: Study: "Everyone knows chocolate is critical to a happy Valentine's Day. Now scientists are one step closer to knowing what makes a heart happy the rest of the year.

It's a gene called DOT1L, and if you don't have enough of the DOT1L enzyme, you could be at risk for some types of heart disease. These findings from a study led by researchers at the University of North Carolina at Chapel Hill School of Medicine appear in the Feb. 1, 2011 issue of the journal Genes and Development."

Action Duchenne Love Runs – there’s still time to register and be officially timed - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-02-17T08:39:00.002Z

Action Duchenne Love Runs – there’s still time to register and be officially timed - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "We’re in the final few days before the Action Duchenne Love Runs for 2011. It’s still not too late to register your entry for the 10k and get a chip so that your run will be timed. And of course you can just turn up on the day."

Parent Project Muscular Dystrophy: 2011 Advocacy Conference & One Voice Summit

2011-02-17T08:39:00.001Z

Parent Project Muscular Dystrophy: 2011 Advocacy Conference & One Voice Summit: "February 14 will mark the ten-year anniversary of the introduction of the MD-CARE Act, the first muscular dystrophy legislation passed by Congress. PPMD led the charge for this critical piece of legislation, which established the National Institutes of Health (NIH) Muscular Dystrophy Coordinating Committee (MDCC) and the Centers for Disease Control and Prevention (CDC) data collection on muscular dystrophy initiative. The MD-CARE Act also called for the creation of scientific Centers of Excellence, named after the late Senator Paul D. Wellstone, across the country to accelerate research on muscular dystrophy. In 2008, again under the leadership and commitment of PPMD’s advocacy campaign, Congress extended the MD-CARE Act to continue funding vital research."

Research Review No. 28 Some new Drugs. - Karl2346 - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy

2011-02-17T08:39:00.000Z

Research Review No. 28 Some new Drugs. - Karl2346 - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy: "While we are waiting for curative treatments such as Exon-skipping, and other types of gene therapy to become available, it is important to keep the DMD patients as able to maintain as much muscle strength as possible for as long as possible. This is because generally when something in the body is lost it is usually lost for good. Currently only steroids are in use to maintain muscle strength longer, but generally they only delay it for about two years."

We need YOUR help to further our Advocacy efforts in Washington, DC

2011-02-17T08:38:00.001Z

Google Reader: "We need YOUR help to further our Advocacy efforts in Washington, DC
from Fling for Logan by garethmitchelson
Google Reader (27): 'We need YOUR help to further our Advocacy efforts in Washington, DC!'
How can YOU help? We need you to make a phone call and send an email!

ACTION 1. Phone Call: Please take a moment to CALL your Senators and Representatives and urge them to sign onto our multimember letter being circulated by Representative Matsui in the House and Senators Wicker in the Senate.

Wondering what to say when you call your Senators and Congressman?

Dont worry, we have provided you with a script: Senate Phone Script House Phone Script

To look up their phone number click here

ACTION 2. Send Action Alert NOW: Email your Representative and Senators-PPMD is requesting your support by clicking through our email action alert below. It takes two minutes!

This simple one step alert will automatically send our message to your Senators and Representative and will also allow you to forward the message to friends and family who may also want to support our Advocacy Agenda.

Click here to send an email to your members of Congress!"

Duchenne Foundation

2011-02-17T08:38:00.000Z

Duchenne Foundation: "Welcome to the homepage of Duchenne Foundation, the only
national non-profit specifically for Australian families affected
by Duchenne muscular dystrophy."

Son Shine Awards - Duchenne Foundation

2011-02-17T08:37:00.000Z

Son Shine Awards - Duchenne Foundation: "The ‘SonShine’ Achievement Awards are designed specifically to celebrate the achievements and strengths of boys with Duchenne muscular dystrophy. "

Biglycan protein

2010-12-30T18:09:00.001Z

A novel potential therapy based on a natural human protein significantly slows muscle damage and improves function in mice who have the same genetic mutation as boys with the most common form of muscular dystrophy, according to a paper published online Dec. 27 in the Proceedings of the National Academy of Sciences. further details here

MU researcher hopes to protect cardiac muscles for children with DMD

2010-12-07T10:21:00.000Z

MU researcher hopes to protect cardiac muscles for children with DMD: "Gene therapy researcher hopes to protect cardiac muscles for children with deadly disease
Children with Duchenne muscular dystrophy (DMD) face a future of rapidly weakening muscles, which usually leads to death by respiratory or cardiac failure before their 30th birthday. While researchers are hopeful that gene therapy could eventually evolve into an effective treatment, few have targeted the heart of the problem as much as Dongsheng Duan, PhD."

The Eileen McCallum Trust | Facebook

2010-11-09T09:45:00.000Z

The Eileen McCallum Trust | Facebook: "The Eileen McCallum Trust Eileen McCallum will be starting next year's Edinburgh Half Marathon which takes place on Sunday 11th April 2011. The charity has taken 50 places and 16 have been allocated to River City cast and crew including Claire Knight, Billy McElhaney, Lisa Gardner, Gary Lamont, Keira Lucchesi and Andy Clark. To see the full lis..."

Recent AVI-4658 trial results have positive outcome - Duchennepedia - Action Duchenne - Fighting for a cure for muscular dystrophy

2010-11-04T11:58:00.000Z

Recent AVI-4658 trial results have positive outcome - Duchennepedia - Action Duchenne - Fighting for a cure for muscular dystrophy: "Results from the recent AVI-4658 study* have been positive so far and are now awaiting further analysis in America by the sponsor AVI BioPharma. Two families that have participated in the trial in the UK share their experiences."

PTC Therapeutics, Inc. - PIVOTAL DATA PRESENTED AT THE WORLD MUSCLE SOCIETY CONGRESS SUGGEST ATALUREN SLOWS THE LOSS OF WALKING ABILITY IN PATIENTS WITH NONSENSE MUTATION DUCHENNE/BECKER MUSCULAR DYSTROPHY

2010-10-18T09:17:00.000+01:00

PTC Therapeutics, Inc. - PIVOTAL DATA PRESENTED AT THE WORLD MUSCLE SOCIETY CONGRESS SUGGEST ATALUREN SLOWS THE LOSS OF WALKING ABILITY IN PATIENTS WITH NONSENSE MUTATION DUCHENNE/BECKER MUSCULAR DYSTROPHY: "PIVOTAL DATA PRESENTED AT THE WORLD MUSCLE SOCIETY CONGRESS SUGGEST ATALUREN SLOWS THE LOSS OF WALKING ABILITY IN PATIENTS WITH NONSENSE MUTATION DUCHENNE/BECKER MUSCULAR DYSTROPHY"

Kennedy Krieger Institute: Kennedy Krieger Institute Launches First Phase II Clinical Trial of Heart Disease Treatment for Duchenne Muscular Dystrophy

2010-10-15T08:43:00.000+01:00

Kennedy Krieger Institute: Kennedy Krieger Institute Launches First Phase II Clinical Trial of Heart Disease Treatment for Duchenne Muscular Dystrophy: "Kennedy Krieger Institute announced today the launch of a first‐of‐its‐kind, phase II clinical trial to investigate a treatment for heart disease in individuals with Duchenne muscular dystrophy (DMD). Made possible by a $545,000 grant from Pilot Trials Now, an innovative DMD initiative organized and managed by Charley’s Fund Inc. and The Nash Avery Foundation, with additional support from Pfizer Inc., the study will examine whether Revatio (sildenafil) improves cardiac function for those affected by the most common fatal genetic disorder in the world."

Prosensa receives �7.5m milestone payment as part of its program with GlaxoSmithKline in Duchenne Muscular Dystrophy - Prosensa

2010-10-14T08:20:00.000+01:00

Prosensa receives �7.5m milestone payment as part of its program with GlaxoSmithKline in Duchenne Muscular Dystrophy - Prosensa: "GSK2402968 (PRO051) is an investigational antisense oligonucleotide which induces exon�skipping of exon 51. The six-month data from the open label Phase IIa extension study in 12�patients with DMD, receiving a weekly 6mg/kg systemic delivery by subcutaneous injection, will�be presented at the 15th International Congress of the World Muscle Society in Japan, October�12-16. The data will be presented by Dr. Nathalie Goemans, MD, from the Department of�Pediatric Neurology, University of Leuven, Belgium."

Acceleron Presents Preliminary ACE-031 Results from a Phase 1 Multiple Ascending Dose Study in Healthy Volunteers

2010-10-14T08:18:00.001+01:00

Acceleron Presents Preliminary ACE-031 Results from a Phase 1 Multiple Ascending Dose Study in Healthy Volunteers: "“The preliminary results of this multiple dose study in healthy volunteers confirm and expand upon the encouraging results observed previously in the single dose study of ACE-031,” said Matthew Sherman, MD, Chief Medical Officer of Acceleron. “These data are encouraging as we continue the development of ACE-031 in the ongoing Phase 2 study in patients with�Duchenne Muscular Dystrophy.”"

Beaker

2010-10-11T13:01:00.000+01:00

Beaker: "Dr. Pier Lorenzo Puri, associate professor at Sanford-Burnham and Italy’s Dulbecco Telethon Institute, and colleagues are figuring out ways to keep the muscle stem cell pool fresh and ready to regenerate injured or diseased muscle. In a study published today in the journal Cell Stem Cell, they uncover the molecular messengers that translate inflammatory signals into the genetic changes that tell muscle stem cells to differentiate. These findings give the scientists a target to artificially dial the stem cell population up or down, a potential treatment that could�boost muscle regeneration in muscular dystrophy patients.

“Our mission is to improve the lives of these patients and extend their lives until they can benefit from a cure 20 years from now,” says Dr. Puri,� a medical doctor who has worked with many muscular dystrophy patients throughout his career."

Antibody that acts as a decoy for mysotatin and binds it before it starts muscle growth inhibition. - Duchennepedia - Action Duchenne - Fighting for a cure for muscular dystrophy

2010-10-11T11:20:00.000+01:00

Antibody that acts as a decoy for mysotatin and binds it before it starts muscle growth inhibition. - Duchennepedia - Action Duchenne - Fighting for a cure for muscular dystrophy: "CAMBRIDGE, Mass. – May 5, 2010 – Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced the initiation of a Phase 2 clinical trial with ACE-031 in patients with Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease. ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling of a cell surface receptor called the activin receptor type IIB (ActRIIB)."

NIH awards three grants to explore novel treatment strategies for muscular dystrophy

2010-09-30T12:30:00.000+01:00

NIH awards three grants to explore novel treatment strategies for muscular dystrophy: "Three grants totaling more than $4.5 million, from agencies of the National Institutes of Health, will be used to explore novel treatment strategies for muscular dystrophy.

The grants were awarded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), National Institute of Neurological Disorders and Stroke (NINDS) and Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) for year one of five-year cooperative agreements."

SOBHD Charity Concert

2010-09-22T08:59:00.001+01:00

The Scottish Official Board of Highland Dancing presents Assemble and Leap: Celebrating 60 years of the Scottish Official Board of Highland Dancing, in aid of Action Duchenne and the MS Society Scotland.

Join a stunning cast of regional, national and international champions, including multiple Scottish, British, Commonwealth and World Champions for this one off spectacular that will showcase Highland Dancing at its very best!

Featuring new and diverse choreography by some of Scotland's most successful highland dance teachers as well as exciting musical performances from world class pipe bands The House of Edgar Shotts and Dykehead and Scottish Power, this is a truly extraordinary event that you don't want to miss!

Tickets are priced at £12.50 and are available from Rachel McLagan and Gemma Baillie, or at www.ticketmaster.co.uk. All proceeds will be divided equally between Action Duchenne and the MS Society Scotland.

Brown grants Tivorsan exclusive license for potential muscular dystrophy treatment

2010-09-22T08:20:00.000+01:00

Brown grants Tivorsan exclusive license for potential muscular dystrophy treatment: "Development of a potential treatment for the most common form of muscular dystrophy is moving into a new phase at Brown University. Brown has granted an exclusive license for the intellectual property to Tivorsan Pharmaceuticals, a startup firm that will strive to see it through human trials.

Officials from Tivorsan and Brown will meet on campus today (Monday, Sept. 20, 2010) to mark the start of this next phase of the potential treatment's development."

Harbor Springs guys Highland Fling for Logan

2010-09-15T09:19:00.000+01:00

Just a quick thank you the the guys of Harbor Springs for their Fling for Logan.

I have attached a picture of Doug's leap - (Doug - I don't know who you are but WOW - boy can you leap) - other pictures are on the flingforlogan website.

Thanks again

Gareth

Christmas fundraising

2010-09-13T15:09:00.000+01:00

Now that it is nearly time to start Christmas Shopping just a wee reminder that you can help raise funds for Logan when you buy online (UK only!).

If you register with our easyfundraising site with Fling for Logan as your chosen cause, then just by clicking the website before you buy (check for instructions) a percentage of the value of your online purchases with participating online retailers (pretty much everyone I could think of) goes to Logan (directly from the retainer).

If you have any questions please email me.

Gareth

NEW: The Eileen McCallum Trust | About the Trust

2010-09-13T13:37:00.000+01:00

The Eileen McCallum Trust | About the Trust: "The Eileen McCallum Trust is a voluntary organisation set up by families in Scotland who are affected by Duchenne muscular dystrophy.

The aim of trust is to provide financial support to families affected by DMD to ensure their sons have the equipment and care services they require for an improved quality of life.

Being a voluntary organisation, all funds raised go directly towards those who require it, boys and young men who have been unfortunate enough to be born with such a devastating condition."

Professor Dame Kay Davies on Biomarin Trial - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

2010-09-13T10:12:00.000+01:00

Professor Dame Kay Davies on Biomarin Trial - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "'The failure of BM195 Phase I trials is obviously very disappointing as it looked so promising in the mdx mice. However, we have new screens coming along which should provide new and better candidates for increasing levels of utrophin. We, like BioMarin, remain committed to utrophin upregulation for the therapy of DMD.'"

Shire announces expansion of Human Genetic Therapies pipeline

2010-09-10T12:17:00.000+01:00

Shire announces expansion of Human Genetic Therapies pipeline: "Shire and Acceleron will jointly collaborate on a worldwide development program to advance ACE-031 into a global Phase 2/3 clinical program designed to demonstrate disease modification in DMD patients. Shire will utilize its Lexington, Massachusetts manufacturing facility to produce commercial supplies of the product for both parties. If marketing authorization is received, Acceleron will commercialize ACE-031 in the U.S. and Canada, and Shire has the exclusive right to commercialize the therapy in the rest of the world."

Acceleron Pharma and Shire announce joint development of ACE-031 for DMD

2010-09-09T14:18:00.000+01:00

Acceleron Pharma and Shire announce joint development of ACE-031 for DMD: "Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, and Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today announced a joint development and commercialization agreement for ACE-031 and other novel molecules targeting the activin receptor type IIB (ActRIIB) pathway. This pathway plays critical roles in regulating the growth of skeletal muscle."

Parent Project Muscular Dystrophy:

2010-09-09T09:31:00.000+01:00

Parent Project Muscular Dystrophy:: "Summary of Clinical Trial Presentations from PPMD's Annual Connect Conference
PPMD's Senior Director of Research and Advocacy, Sharon Hesterlee, has written up a detailed summary of the presentations from this summer's Connect Conference regarding clinical trials - one of our most popular sessions of the Conference. PowerPoint presentations from the presenters are available for download, for these presentations, as well as many other Conference sessions"

Progress - slow and painful

2010-09-06T13:51:00.000+01:00

On 2 August BioMarin announced that they were stopping the development of their Utrophin upregulation drug.

Utrophin, from what I can determine, is produced by the body when an infant and performs a similar role to that of dystrophin. As the child grows older the body then switches to producing the more effective dystrophin.

The aim behind this research was to get the body to start producing Utrophin in sufficient quantities to compensate, to some degree, from the lack of Dystrophin produced by the boys.

BioMarin has not stopped all their work - merely this particular process - and so whilst it is not the end of the line it is still a bit disappointing and another reminder of how slow and painful progress can be.

http://www.actionduchenne.org/viewarticle?news=90

International study to improve treatments for Duchenne muscular dystrophy launched

2010-09-03T16:30:00.000+01:00

International study to improve treatments for Duchenne muscular dystrophy launched: "A large international study aimed at improving the care of muscular dystrophy patients worldwide is being launched by physicians, physical therapists, and researchers at the University of Rochester Medical Center."

Nationwide Children's Hospital designated as Wellstone Muscular Dystrophy Cooperative Research Center

2010-08-30T13:38:00.000+01:00

Nationwide Children's Hospital designated as Wellstone Muscular Dystrophy Cooperative Research Center: "A $7 million grant from the National Institutes of Health (NIH) will help researchers at Nationwide Children's Hospital translate new scientific findings and technological developments into novel treatments for the muscular dystrophies. The grant designates Nationwide Children's Hospital as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, one of three national award recipients in 2010."

Google Reader (1)

2010-08-19T15:11:00.000+01:00

Google Reader (1): "CAMBRIDGE, Mass. -- Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced it received FDA Fast Track designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients suffer from the progressive loss of muscle mass and strength."

Parent Project Muscular Dystrophy: Coach to Cure MD

2010-08-18T08:18:00.000+01:00

Parent Project Muscular Dystrophy:: "Today is a critical day for Duchenne. Actually every day is a critical day for Duchenne. And we know that often it may seem we are coming to you, the Duchenne community, to get involved and to take part in numerous things. That is because no one can do everything...We need your help in this fight to end Duchenne. We need you to join Coach To Cure MD.

By joining our team, by participating in even the smallest way, you help:

push critical research forward;
develop standards of care to ensure that your child is cared for properly; and
remind decision-makers in Washington that our boys count - that their lives matter.
Participating doesn't have to hard, it doesn't have to be time consuming. But we must work as a team if we are going to see success in our sons' lifetimes."

Acceleron's ACE-031 protein therapeutic for DMD receives FDA Fast Track designation

2010-08-05T08:35:00.000+01:00

Acceleron's ACE-031 protein therapeutic for DMD receives FDA Fast Track designation: "Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced it received FDA Fast Track designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients suffer from the progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength."

Rachel McLean - still on top of the world!

2010-07-20T09:05:00.000+01:00

Definitive Highland Dancing Resource - toeandheel.com: "On Saturday Rachel danced an astonishing Fling on top of Ben Nevis.

Amazing enough to have the energy to climb the mountain, never mind getting changed whilst exposed to the elements and then dancing on a wet, slippery, cold stone slab, at the edge of a precipice, in 50 mph winds, in the cold, damp & wet conditions, in near nil visibility . . . .

Rachel is certainly still on top of the World."