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Katie Brooks is a mom of two incredible little boys, Dominic and Daniel. She was born and raised in Austin, TX and moved to Atchison, KS to attend college at Benedictine…

A recent 60 Minutes segment examining the cost of gene therapy put a national spotlight on a question families living with rare disease confront every day: how can lifesaving medical…

Clinical trials are key to advancing research for neuromuscular disease treatment. Here’s what you need to know why to get involved and how to enroll.

Stepping into this role in such a historic year has been humbling and inspiring. This is my first End of Year message as MDA’s President and CEO, and I want…

The idiopathic inflammatory myopathies (IIMs) are a group of rare autoimmune muscle diseases that include dermatomyositis, polymyositis, immune-mediated necrotizing myopathy (IMNM), antisynthetase syndrome, and inclusion body myositis (IBM). These disorders…

Researchers at Sanofi are seeking individuals with non-congenital myotonic dystrophy type 1 (DM1) to participate in a phase 1/2 clinical trial to evaluate the safety of an investigational gene therapy (SAR446268)…

The last 12 months have been full of twists and turns in Washington D.C., but through all the unexpected events, MDA’s advocates remained steadfast and ensured that their voices were…

Researchers at Genentech Inc. are seeking pediatric patients previously treated with gene therapy (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) to participate in either of the two interventional studies (HINALEA 1…

Researchers at Dyne Therapeutics, Inc. are seeking individuals with myotonic dystrophy type 1 (DM1) to participate in a phase 1/2 clinical trial (ACHIEVE) to evaluate the safety and efficacy of the…

When the disability rights movement began in the 1970s, the disability community started forging the path for those living with disabilities to have access to equal rights, inclusion, and the…