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Clinical Research Alert: Real World Study of Male Fertility Following Risdiplam Treatment
Researchers at Genentech Inc. are seeking adult males treated with risdiplam (Evrysdi) for spinal muscular atrophy
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Quick Guide: Understanding Programs and Benefits for People with Disabilities
Along with the daily challenges of living with a disability, many individuals face the added
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Becker Muscular Dystrophy Series – Part 2: Cardiopulmonary Care
The second session in PPMD’s Becker Muscular Dystrophy webinar series focused on cardiopulmonary care. Our
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Tis the Season for Holiday Hacks and Tips
For some, the holiday season means the hustle and bustle of hosting, traveling, gift giving,
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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
