Catalyst Pharmaceuticals has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug amifampridine phosphate (brand name Firdapse) for the treatment of people with Lambert-Eaton myasthenic syndrome (LEMS). The FDA has 60 days to determine whether the NDA is complete and acceptable for filing. If the FDA . . .
MDA’s 2018 Clinical Conference is the preeminent gathering of MDA Care Center health care providers and other medical and scientific experts from across the country designed to enhance the communication of new medical advances and information relating to the delivery of best-in-class care for individuals with neuromuscular disease.
In 2017, MDA helped thousands of families live better and longer lives. All of this and much more was possible thanks to you. Thank you for helping MDA make 2017 a remarkable year for the families we serve.
James Lupski, who lives with Charcot-Marie-Tooth disease type 4B., was awarded an MDA research grant totaling $300,000.
Andrew Boychuk, who lives with LGMD, shares the positive impact MDA’s LGMD genetic testing program has made in his life.
Elamipretide, which treats muscle weakness caused by mitochondrial disease, has received FDA Orphan Drug Designation
Do you or a loved one live with LGMD? Learn more about MDA’s LGMD genetic testing program and find out how to make an impact on LGMD research.
Ralph Yaniz foudn himself on the cutting edge, literally, of LGMD research when he donated his muscle and skin cells.
Sarepta announced encouraging results from a phase 1/2 DMD clinical trial for golodirsenm which targets exon 53.
Monkol Lek, a researcher at Massachusetts General Hospital and the Broad Institute, both lives with and studies limb girdle muscular dystrophy. He received a research grant from MDA to improve the diagnosis of rare muscle diseases, and this fall he will open his own lab at Yale.
