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How it feels to get recognition for my life as a caregiver
I’m no longer a young mom, but I was once. In fact, I was a […]
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Watch: Capricor Therapeutics – Regulatory Update & Clinical Insights on Deramiocel for Duchenne Cardiomyopathy
Capricor Therapeutics recently joined PPMD for a community webinar on Tuesday, July 29, 2025 to […]
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Duchenne MD gene-editing therapy nets orphan drug designation
An experimental gene editing therapy for Duchenne muscular dystrophy (DMD) being developed by Precision Biosciences […]
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Community Voice: Why We Decided to Name MDA in Our Wills
Probably like you, recent years have altered our lives in ways both expected and completely […]
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Behind the Drug: Risdiplam (Evrysdi) for SMA
Spinal muscular atrophy (SMA) is a rare genetic disease affecting 1 in 11,000 live births […]
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A weekend trip with my family stirred up mixed feelings
Last summer, I wrote a column about my beach vacation with my family. It was […]
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Your Voice Is Everything
When the Duchenne and Becker community speaks, the world listens — not just because of […]
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Elevidys shipments for ambulatory DMD patients to resume
Sarepta Therapeutics will soon resume shipping Elevidys (delandistrogene moxeparvovec-rokl) for ambulatory Duchenne muscular dystrophy (DMD) patients, […]
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Sarepta to Resume ELEVIDYS Shipments to Ambulatory Patients After FDA Recommends Removal of Voluntary Hold for Ambulatory Population
The U.S. Food and Drug Administration (FDA) has announced that the agency is now recommending […]
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Simply Stated: Updates on Friedreich’s Ataxia (FRDA)
Friedreich’s ataxia (FRDA) is an inherited neuromuscular disease that primarily impacts the nervous system and […]
