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MDA Scientific Conference To Focus On Therapy Development For Neuromuscular Diseases

TUCSON, Ariz., April 22, 2013 /PRNewswire-USNewswire/ -- Some 500 members of the scientific and medical research community are scheduled to attend the 2013 MDA Scientific Conference, "Therapy Development for Neuromuscular Diseases: Translating Hope into Promise," April 21-24 in Washington, D.C., according to  representatives of the Muscular Dystrophy Association (MDA). MDA organizes the biennial conference.

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The conference will allow experts from various backgrounds — including the academic, corporate drug development and clinical arenas — to focus on therapeutic strategies for neuromuscular diseases and discuss how barriers to therapy development may be identified and overcome. 

Different aspects of therapy development, including novel targets, biomarkers, therapeutic modalities and new animal models, will be highlighted during the two-and-a-half day event, which will include more than 60 platform presentations and a poster session with an estimated 200 posters. A specially designed phone app will be available to help attendees optimize their time during the jam-packed conference.

"This conference offers a tremendous opportunity for professionals from different disciplines to share perspectives and bring each other up to date on the latest advances and techniques in neuromuscular disease research," said Frank Bennett , Ph.D., senior vice president of Isis Pharmaceuticals, who is serving as a co-chair of the conference.  Isis is currently conducting trials of potential antisense therapies for amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA); a report on the potential SMA therapy, ISIS-SMNRx, is on the conference agenda. 

Bennett added: "MDA is to be lauded for its leadership role in supporting and partnering with diverse players in the neuromuscular arena, each of whom brings unique wisdom and experience to the challenging task of developing effective therapies."

Therapy development for SMA, ALS, facioscapulohumeral muscular dystrophy, myotonic dystrophy, Duchenne muscular dystrophy, Becker muscular dystrophy, and other progressive and potentially life-threatening neuromuscular diseases will be under discussion.

Up-to-the-minute news to be presented at the conference also will include reports from:  

  • Massachusetts-based Sarepta Therapeutics (exon skipping, eteplirsen, Duchenne dystrophy);
  • London-headquartered GlaxoSmithKline (GSK), in collaboration with Dutch-based Prosensa (exon skipping, drisapersen, Duchenne MD);
  • San Francisco's Cytokinetics (troponin activator, tirasemtiv, amyotrophic lateral sclerosis);
  • Massachusetts-headquartered Repligen (HDAC inhibitors, Friedreich's ataxia); and
  • MDA-funded trial (lisinopril and losartan, cardiomyopathy in Duchenne MD).

Jerry Mendell , a longtime MDA research grantee and co-director of the MDA Clinic at Nationwide Children's Hospital in Columbus, Ohio, is lead investigator in Sarepta's trial of the exon-skipping drug eteplirsen. On Sarepta's behalf, Mendell will report on the 74-week results of its phase 2b eteplirsen trial in 12 boys with Duchenne MD.

Thomas Voit , medical and scientific director of the Institut de Myologie in Paris, will offer data on behalf of GSK/Prosensa from the company's phase 2b study of its own exon-skipping compound for Duchenne, drisapersen. 

Eric Hoffman , Ph.D., director of the Research Center for Genetic Medicine at Children's National Medical Center in Washington, D.C., is serving with Frank Bennett as co-chair of the 2013 conference. Hoffman, a longtime MDA research grantee, was a key investigator in the identification of the role of the dystrophin protein in Duchenne and Becker muscular dystrophies, and is currently studying asynchronous repair in Duchenne.

"This is an extremely promising moment in neuromuscular science, with more clinical trials of potential therapies under way than at any time in history," Hoffman said. "I can say with confidence that if it were not for MDA's fostering of innovative science and its sponsoring of conferences such as this one, prospects for families affected by neuromuscular diseases would be much less hopeful."

MDA's first national-scale scientific conference took place in Las Vegas in March, 2011, with 300 people attending. The biennial scientific gatherings take place in odd-numbered years, with MDA conferences emphasizing clinical modalities held in even-numbered years. 

Biopharmaceutical companies serving as sponsors of the 2013 MDA Scientific Conference are: Sanofi (platinum level); Biogen Idec and Sarepta (gold level); Catabasis, Cytokinetics, Fate Therapeutics, Pfizer, PTC Therapeutics, Shire and Tivorsan Pharmaceuticals (silver level); and with special thanks going to ARMGO Pharma.

"The progress being made in neuromuscular disease research is truly astonishing, when you think of how far we've come with therapy development compared to where we stood only five years ago," said MDA President and CEO Steven M. Derks . "MDA families are praying and hoping for positive research outcomes leading to new therapies, and that's why I think it's absolutely essential for researchers to come together in a cooperative spirit to aid and inspire each other's efforts as much as possible."

Derks also said: "I'm confident that attendees will come away from this conference not only with fresh perspectives, but with a renewed sense of hope and optimism as we look to even more exciting developments in the months and years ahead."

For more information on the MDA National Scientific Conference, and to read blogs posted during the conference, go to

About MDA
MDA is the nonprofit health agency dedicated to finding treatments and cures for muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education.

In addition to funding more than 250 research projects worldwide, MDA maintains a national network of 200 medical clinics; facilitates hundreds of support groups for families affected by neuromuscular diseases; and provides local summer camp opportunities for thousands of youngsters living with progressive muscle diseases.

For more information, visit and follow MDA on Facebook ( and Twitter (@MDAnews).

SOURCE Muscular Dystrophy Association
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