Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives.
Gene and Cell Therapy Approaches Hold Promise for Usher Syndrome
September 21st 2024In honor of Usher Syndrome Awareness Day, CGTLive® interviewed Zheng-Yi Chen, DPhil, associate professor, Otolaryngology–Head and Neck Surgery, Harvard Medical School, about the current state of research in this rare disease.
Paul Melmeyer, MPP, on Advocacy and Progress in Rare Neuromuscular Diseases From a Policy Standpoint
February 29th 2024In observance of Rare Disease Day 2024, the vice president of public policy and advocacy at the Muscular Dystrophy Association discussed advocacy and policy progress regarding rare neuromuscular diseases.
Sharon Hesterlee, PhD, on Collaborating to Expand Therapy Development to More Dystrophies
November 25th 2023The chief research officer of the Muscular Dystrophy Association discussed the need for disease-modifying therapies in other muscular dystrophies and programs the association has announced to this end.