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MDA 2015 Scientific Conference to Emphasize Urgent Need for Discovery and Treatment

Leading experts in neuromuscular disease research and drug development will present and exchange ideas and information March 11-14

CHICAGO, March 10, 2015 — The Muscular Dystrophy Association will hold its 2015 Scientific Conference at the Washington Marriott Wardman Park in Washington, D.C., March 11-14. The meeting is the preeminent gathering of the nation’s leading scientists, researchers and professionals specializing in neuromuscular disease research and care, designed to accelerate discoveries and drug development for families fighting neuromuscular diseases, for which there are currently no cures. The conference is part of MDA’s annual conference series, which hosts clinical gatherings and scientific research gatherings in alternate years.

“One key strategy to accelerating lifesaving and life-enhancing treatments and cures for neuromuscular and motor neuron diseases is to ensure collaboration among the nation’s top thought leaders in our scientific community,” said Steven M. Derks, MDA President and Chief Executive Officer. “I’m proud that, in addition to the hundreds of different research projects we fund, MDA, through our Scientific and Clinical Conferences and symposia, continues to be a convener and facilitator for researchers nationwide to share ideas, best practices, and novel strategies.  Shared knowledge through scientific discoveries and failures speeds progress for the families we serve. Together, we are accelerating frontline discovery and bringing urgently-needed answers to children and adults affected by muscular dystrophy, ALS and related life-threatening diseases.”

This year’s conference will focus on better understanding disease causes, identifying therapeutic targets and discussing new advances in preclinical and clinical research — all aimed at accelerating drug development and treatments for neuromuscular diseases. The meeting will leverage the expertise of researchers across different neuromuscular disease areas to drive scientific and therapeutic developments.

“MDA has played a role in nearly every neuromuscular disease breakthrough since we started this fight in 1950. This is a promising time in neuromuscular disease research as we are seeing an increase in late-phase clinical trials that could result in treatments for many of our families,” said Valerie A. Cwik, MDA Executive Vice President and Chief Medical and Scientific Officer. “Our conference will be packed with presentations discussing everything from early-stage therapeutic targets through to the most up-to-date news as drugs move through the development pipeline to possible FDA approval.”

More than 400 attendees from academic laboratories, clinics and the biotech and pharmaceutical industry are expected, with more than 40 platform presentations and more than 130 poster presentations planned. Keynote Speaker Nenad Bursac of Duke University will open the conference on Wednesday, March 11. Bursac is an established leader in research focused on cell and tissue replacement therapy who recently reported groundbreaking results demonstrating the ability to grow human tissue in the laboratory that contracts as if it were in the body.

MDA Goodwill Ambassador Kourtney Rivers, and her family, will attend the conference and speak to the group about the role MDA has played in her life, and the urgent need for further scientific advancements. Kourtney was diagnosed with myotonic muscular dystrophy, a form of muscular dystrophy that affects muscles and many other organs in the body, at the age of one. She and her family have been active supporters of MDA in the D.C. area, helping out with MDA events such as MDA Muscle Team, Team Momentum, softball tournaments, and numerous other events throughout the year.

Conference Content

Presentations for neuromuscular disease professionals will focus on the following areas:

• Genetics, epigenetics and genetic modifiers – Is DNA sequence the only part of the story when it comes to health and disease? How epigenetic factors are gaining importance.
• Targets and therapeutic approaches – The last decade has shown significant advancements around the molecular basis of neuromuscular disorders. Today, scientists are starting to put that knowledge to use in designing therapies for these conditions.
• Mechanisms of disease – Understanding how disease mechanisms translate into therapy development is especially important to today’s researchers as therapies are developing faster than ever.
• Animal models, preclinical studies and biomarkers – How are today’s scientists studying animals, cellular models, and biochemical indicators to find safe and effective treatments for neuromuscular disorders?
• Clinical trials and late-breaking news

Meeting Supporters

MDA is deeply grateful to the following companies for their support of the 2015 Scientific Conference:

• Hope level — BioGen Idec
• Platinum level — Sarepta
• Silver level — Catabasis Pharmaceuticals, Inc.
• Silver level — PTC Therapeutics
• Break Session level — ARMGO Pharma, Inc.
• Break Session level — Cytokinetics
• Break Session level — Catalyst
• Other – IOS Press, Inc.

About MDA

The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.

Visit mda.org and follow us at facebook.com/MDAnational and @MDAnews. Learn more about MDA’s mission by watching this video.

Claire Orphan
Media Relations Manager
(312) 260-5928
corphan@mdausa.org