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FDA approves new safety warning and updated prescribing information for Sarepta’s Elevidys
Letter to the community added November 17, 2025 The FDA has approved the new labeling […]
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Insmed’s ASCEND Study and INS1201, an Investigational Gene Therapy for Duchenne Muscular Dystrophy
Watch HERE The post Insmed’s ASCEND Study and INS1201, an Investigational Gene Therapy for Duchenne […]
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FDA authorizes a new liver safety biomarker relevant for Duchenne and Becker
We congratulate C-PATH (Critical Path Institute) on their success in getting the FDA to qualify […]
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New FDA Elevidys label limits use to DMD patients who can walk
The U.S. Food and Drug Administration (FDA) has approved an update to the prescribing information […]
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5 Things Advocates Should Know: The Alleviating Barriers for Caregivers Act
November is National Family Caregivers Month, and it is important to MDA to uplift the […]
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Insmed’s ASCEND Study and INS1201, an Investigational Gene Therapy for Duchenne Muscular Dystrophy
Watch Webinar HERE The post Insmed’s ASCEND Study and INS1201, an Investigational Gene Therapy for […]
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Progress Now: Immunotherapy Study Recruiting, Drug Cleared for Expanded Access, and More
Amyotrophic lateral sclerosis (ALS) Immunotherapy Study Recruiting Coya Therapeutics has launched the phase 2 ALSTARS […]
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New Year, New Financial Plan: Tips to Get Your Budget Back On Track
For many families living with neuromuscular diseases, balancing health and disability-related expenses with rising household […]
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Understanding Spinal-Bulbar Muscular Atrophy
Spinal-bulbar muscular atrophy (SBMA) is a genetic disorder that affects muscles and nerves, leading to […]
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From Classroom to Courtroom: How Eric Arnold Navigated Accessibility in His Education and Career
Eric Arnold has built his life around sharing his skills and expertise with others — […]
