Stay informed. See MDA updates on COVID-19

Innovations in Science

We're accelerating the delivery of treatments and cures.

What We've Achieved

MDA is the nation’s largest nonprofit supporter of research on over 40 different neuromuscular diseases for over 65 years. Every year MDA supports hundreds and hundreds of physicians and scientists in the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. As such, MDA has contributed to many major advances in muscle biology and therapy development over the decades.

MDA sponsored research has resulted in breakthroughs in treating diseases. For example, MDA-funded research helped Genzyme develop a therapy for Pompe disease called Myozyme, which has saved the lives of many patients with this devastating disease. Even where no “cure” is available yet, MDA research has resulted in better treatments that result in increased survival and better quality of life. In the past, boys with Duchenne muscular dystrophy (DMD) died in their teens, but there are now some 40-year-old men living with the disease.

MDA has been involved in research into basic muscle and nerve biology since MDA’s inception, when virtually nothing was known about how muscles were formed or functioned, even in non-diseased tissues. Since then, thousands of scientific papers have been published explaining how the tissues work and what goes wrong in neuromuscular disease. MDA funding supported the research that led to the discovery of the genetic causes of dozens of diseases, starting with the discovery of the dystrophin gene in 1986. Without this knowledge, there was no hope for curing diseases. While continuing to make new discoveries in this area, this research is also paying dividends as new therapies move into clinical testing and to market.

MDA’s research program has been so successful over the years that MDA started the translational research program in 2004 to help accelerate therapy development based on these results. This program attracted so much interest that it was expanded in 2009 by the formation of MDA Venture Philanthropy (MVP), which operates with a more venture capital-like approach. MVP is exclusively focused on the funding and commercialization of treatments and cures for neuromuscular diseases, and hopes to increase the speed of moving drugs through clinical development.