Biglycan protein

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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:

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

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

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

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)

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

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

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!

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."

Rachel's Fling on Ben nevis

Rachel's McLean's astonishing Fling on the top of Ben Nevis.

Rachel's aunt (Catriona) was there and said

'Rachel was a wee star on Sat although my heart was in my mouth as the winds were quite bad ( 50 mile an hour reported!) and I was just waiting for her to blown off!! She was getting dangerously near the edge a couple of times, probably my shrieks didn't help!'

What an astonishing Fling!

Australia's Fling for Logan - email from MD Queensland

I have copied below an email to Margaret Johnston (the organiser of the Australian Fling for Logan) from Rick Carr of Muscular Dystrophy Queensland.

Hello Margaret,

It was so nice to meet you and the rest of the committee at the meeting last night.  Thank you so much for your kindness, and generosity.

We are preparing the receipt for your donation, would you be able to provide me with the postal address of your organisation so we can forward this to you?

Logan’s story will stick in my mind, as will the sight of those 412 dancers doing the Highland Fling!

Warm Regards,

Rick Carr

CEO

Muscular Dystrophy Queensland

Congratulations Rachel

Just got a text from Rachel - she got to the top of Ben Nevis and completed her Fling.

The weather was terrible and she had no signal at the top and so had to wait until half way back down to text me.

She has the fling on video - I will upload it later this week.

Congratulations again and many thanks.

Gareth

Denver Mayor proclaims June 26 "End Duchenne Day"

Middletown, OH – June 18, 2009 – 

Patricia A. Furlong, Founding President and CEO ofParent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced that the Mayor of Denver, Colorado, John W. Hickenlooper, has proclaimed June 26, 2010 “End Duchenne Day” in recognition of PPMD’s 16th Annual Connect Conference. Denver is playing host to the country’s largest Duchenne-specific, international conference, June 24 – 27, 2010.

Rachel's Fling on Ben Nevis

On July 17 this year Rachel McLean will be attempting to carry out a sponsored Fling for Logan challenge.

Aiming high is nothing new for Rachel. A relatively late starter to Highland Dancing she rapidly progressed through the ranks to reach the peak in 2004 when she was crowned World Highland Dancing Champion.

However, this challenge will see Rachel climb to new heights - she has decided to climb Scotland's Highest Mountain - Ben Nevis - and plans to perform her Highland Fling on the summit!

Not content with miles to treck and hill to climb - she will need to carry her music and kilt outfit to the top with her.

You can follow Rachel's progess on the blog and sponsor her on the sponsor page.

Australias Fling for Logan

At last I have had a chance to upload video footage from the fling for Logan organised by South Queensland Region Highland Dancing inc.

Having just finished watching it I can only say again many, many thanks to all those involved in it - the organisers, the dancers (all 411 of them) and not least the piper who had to play them all into place.

Gareth

Marathon's map

I have now included an events map in the flingforlogan.com website.

This shows the locations (and dates) of the various fundraising events that are coming up - for now it lists the forthcoming marathons and half-marathons in the UK and US that can be run for Duchenne.

Please checkout the website for details.

Run for our sons

Parent Project Muscular Dystrophy (the US Charity fighting to end Duchenne) has a 'run for our sons' fundraising campaign.

If you live in the US and want to find out how you can get involved - visit the parentprojectMD.org website for details.

Upcoming events include:

June 26           Seattle 1/2 marathon
July 11            Missoula 1/2 marathon
September 5   Disneyland 1/2 marathon
October 10    Chicago marathon
October 17    Denver marathon
November 7  New York marathon

Forthcoming Events

Are you a runner?

Would you consider running for Action Duchenne?

The following races are coming up and can all be run on behalf of the Charity:

May 23           Edinburgh Marathon
May 31           BUPA 10k (London)
July 3              Birmingham & Black Country 1/2 marathon
September 5    Bristol 1/2 Marathon
September 12  Robin Hood Run (Nottingham)
October 11     Cardiff 1/2 marathon

Check out the Action Duchenne events page for contact details.

Australia's Fling for Logan

I have just had an email from Margaret Johnston updating me on the Fling for Logan that was held on the weekend of the Champion of Champions in Australia.

I do not know yet hoe many dancers too part but it seems that a great time was had by all the competitors (and parents) who took part.

Hopefully I will have some details to post soon.

Anyway, many thanks to all who participated and helped further raise awareness of the Duchenne condition in Australia and further afield.

Gareth

New Action Duchenne Cook-Book

Action Duchenne launched its new cook-book 'Looks Good, Tastes Great - Jewish Culinary Favourtes' last month and it has already received great reviews.

The cook book features recipes from celebrities and well known personalities in the Jewish community, including Nigella Lawson, Michael Grade, Maureen Lipman, Ed Miliband, Michael Winner and David Pleat. All proceeds from the book will raise money for the charity.

Buy the book direct from Action Duchenne by clicking here

Australia's biggest Highland Fling

Easter brings the skirl of the bagpipes and the swing of the kilt to Caloundra on Queensland’s’ Sunshine Coast with both the Australian Pipe Band Championships on Easter Saturday and Highland Dancing Championships on Easter Saturday and Sunday.

Also happening is an attempt at Australia's biggest Highland Fling - a special event being organised to raise awareness of Duchenne Muscular Dystrophy.

For details - contact Margaret

Funds raised are being allocated to Logan and also the Duchenne organisation in Australia.

Best of luck to all the dancers.

£2,065 for Action Duchenne

We are delighted to announce that the profits from the charity competition, including the raffles, canteen, sales of tablet and guess the bear's birthday came to an astonishing £2,065.

A cheque for this has been sent to Deann Widd of the charity who was overwhelmed by the whole event.

He said that 'the Charity is delighted to receive the cheque, which far exceeded all expectations, and that he personally was amazed by the support given by the dancers and their families.

Thanks to all

Donna & Gareth

Concert for Duchenne & Multiple Sclerosis

Last week I received a letter from Rachel McLaggan & Gemma Baillie informing me that a charity concert will be held later this year.

Proceeds will be split between Action Duchenne & UK Multiple Sclerosis.

Date & Venue - 6 November 2010, Alhambra Theatre, Dunfermline

The concert is being timed to conincide with the 60th anniversary of the SOBHD.

Further details will be posted when they become available.

Gemma's 5K

Last December Gemma (Baillie) traded her kilt outfit for a penguin costume as she undertook a 5K run with Duchenne as her chosen charity.

It looked way too cold to be outside but Gemma ran on despite the penguin conditions - well done Gemma.

(I have added some other pics from her run to the photo page.)

website freshened up

After a couple of (very) long nights I have at last finished freshening up the flingforlogan.com website.

New layout & new content - including everypage showing my latest blog entries & tweets.

UK Government petition

A petition on the UK Prime Minister to put more funds into Duchenne research has been setup.

Any UK citizen may complete it by clicking here.

Please help us put pressure on the Prime Minister and sign up.

Gareth

Laura's Skydive

Laura will be undertaking a sponsored Skydive to raise funds for Logan.

Exact date not yet finalised (she is waiting to find a date that won't clash with the highland games) but target is June or July.

Best of luck Laura

Guess the bear's Birthday

Only 15 days to go . . . .

£5 per guess - buy online here

Australia's Largest Highland Fling

The date has been set for what will hopefully be Australia's largest Highland Fling.

The challenge, being run in conjunction with the Champion of Champions of Australia Championship, which will raise funds for Logan and also Duchenne research, will take place on 4 April 2010.

Details to follow soon.

New photo uploaded

I have just received some photos from the South African OBHD re the fling that they organised last summer for Logan.

I have uploaded 1 photo the the photo file and will hopefully add more when these are received.

Guess the bear's birthday

The teddy in the photo is being raffled to raise funds for Action Duchenne (the Charity)

Guess the bear's birthday - £5 per guess -

click here to guess

New photos

Some new photos uploaded