At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. 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.
MDA is pursuing the full spectrum of research approaches toward combating neuromuscular diseases, from fundamental discoveries of the causes of disease to clinical trials of potential treatments. MDA also helps spread this 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.
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.
Investigators seek participants for a phase 2 clinical trial to test CK-2127107 in spinal muscular atrophy. The investigational drug may enhance muscle function in people with SMA.
Read questions and answers about the FDA and its process for approving drugs.
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.
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