Sarepta Therapeutics is developing eteplirsen, an experimental exon-skipping drug for Duchenne muscular dystrophy (DMD). Later this year, the Cambridge, Mass., biopharmaceutical company expects to determine the regulatory pathway for eteplirsen, including the feasibility of an application for accelerated approval, and is moving forward with plans for an additional clinical study to begin in early 2014.
DMD affects one out of every 3,500 newborn boys, and MDA has supported the development of exon skipping through its basic research program since the 1990s.
“Our company is proud to support the MDA Scientific Conference, which provides leading academics and clinicians from across the country with the opportunity to exchange ideas and share perspectives on the latest developments and trends in neuromuscular medicine,” said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. “As a community, we are on the threshold of achieving major advancements in the understanding and treatment of neuromuscular diseases such as DMD, thanks in great part to MDA and its support for research over many years.”
About 500 scientists, many of whom are at the vanguard of neuromuscular disease research, will be attending the conference and presenting their newest findings about Duchenne muscular dystrophy, spinal muscular atrophy, ALS and additional neuromuscular diseases. Fueled by rapidly evolving biotechnology practices, an unprecedented number of new potential disease therapies have been developed.
“Five years ago, this meeting couldn’t have happened," said MDA Vice President of Research Jane Larkindale. "Advances in biotech research are moving quickly, and we now have promising new paths for treatment development such as exon skipping in DMD. Our conference highlights projects from early-stage therapeutic targets through to clinical trial results. MDA appreciates the support Sarepta is providing for this conference and for disease research. MDA has been funding muscle disease research for more than 60 years, and the progress that Sarepta is making is encouraging to us and to MDA families.”
In addition to the academic scientists who will be attending the meeting, MDA is bringing together professionals from government, industry and the nonprofit sector to join the discussion about therapy development for neuromuscular disorders.
Conference co-chairs are: C. Frank Bennett, senior vice president for research at Isis Pharmaceuticals; and Eric Hoffman, director of the Research Center for Genetic Medicine at Children's National Medical Center in Washington, D.C.
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.