MDA is the world’s largest nonprofit supporter of research on neuromuscular diseases. MDA supports nearly 350 physicians and scientists each year, with a research commitment in about 40 different neuromuscular diseases of about 30 percent of the total amount spent by the National Institutes of Health. As such, MDA has contributed to almost every development in muscle biology in the last 50 years.
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.
To learn more about how far MDA has come in its research, visit State of the Science and select the diseases provided in the list.