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On Mar. 5, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug designation to Acceleron Pharma’s ACE-083, a locally acting muscle agent, for treating Charcot-Marie-Tooth disease (CMT). Delivered by…

A young man with SMA answers this question by becoming a student leader

A Q&A with researcher Stephen Meriney, Ph.D.

Think outside the cubicle to find solutions to common accessibility challenges in the office

In the face of the everchanging medical landscape, it is important that you prepare yourself and family members to go to the emergency room alone or without the option of…

When neuromuscular disease impacts your ability to perform daily tasks, cultivating problem-solving skills and support can help you live independently in your home

Researchers at Neurogene are seeking participants living with Charcot-Marie-Tooth disease (CMT) type 4J to participate in a natural history study. This study aims to better understand disease course so researchers will be able…

On Feb. 27, Sarepta Therapeutics announced positive interim results of a phase 1/2a clinical trial for MYO-101, a gene therapy candidate developed by Myonexus Therapeutics for patients living with limb-girdle…

It’s March, and at MDA that means only one thing: It’s Shamrocks season! Each St. Patrick’s Day season for the last 37 years, MDA has partnered with 20,000 retailers nationwide…

Last week, Sen. Tammy Baldwin of Wisconsin introduced the Air Carrier Access Amendments Act (S. 669). MDA commends Sen. Baldwin for championing this legislation that sets forth important protections for…