We're taking a big picture perspective across neuromuscular diseases to uncover breakthroughs for treatments and cures.IMPACT IN 2016
The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families. We also help spread scientific knowledge and train the next generation of scientific leaders by funding national and international research conferences, clinical research training grants and career development grants.
The FDA has granted accelerated approval to eteplirsen for the treatment of Duchenne muscular dystrophy! Decades of MDA research and investment have paid off with the first disease-modifying drug available to treat DMD.
We’re taking a big picture approach to accelerating treatments and cures for muscular dystrophy, ALS and related diseases.
Phase 1 trial, sponsored by Biogen and Ionis Pharmaceuticals will test Ionis-SOD1-Rx in people with ALS.
MDA’s chief medical and scientific officer expresses optimism that the drug eteplirsen could change the course of Duchenne muscular dystrophy and be the first of what MDA hopes will be many new treatments for MDA families.
PTC Therapeutics is working with the FDA to discuss and clarify concerns about its New Drug Application for its experimental DMD treatment.
Antoni Barrientos is working to address a knowledge gap about mitochondrial protein complex assembly defects, which are a frequent cause of inherited mitochondrial myopathies.
MDA research staff oversee grants programs and provide guidance on scientific and medical matters for MDA. Staff members interact with federal agencies, international neuromuscular disease partners, drug development groups and pharmaceutical companies, as well as with patient advocacy groups and other neuromuscular disease stakeholders. In addition, they manage basic and translational research portfolios relating to the diseases in MDA's program.
Our trained specialists are here to provide one-on-one support for every part of your journey. Send a message below or call us at 800-572-1717. If you live outside the U.S., we may be able to connect you to muscular dystrophy groups in your area, but MDA services are only available in the U.S.