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DYNE-251 for DMD granted FDA breakthrough therapy designation
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to Dyne Therapeutics’ […]
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Dyne Therapeutics granted Breakthrough Therapy Designation for exon-51skipping therapeutic, DYNE-251
CureDuchenne was an early investor in Dyne Therapeutics in 2020, and congratulates the company for receiving Breakthrough Therapy […]
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Dyne Therapeutics, Inc. announced today FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy
Dyne Therapeutics has announced FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy. This […]
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MDA Ambassador Guest Blog: Built to Rise – Strength Forged Through Every Challenge
Darlene, who was diagnosed with spinal muscular atrophy (SMA) type 3 at age 19, just […]
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Clinical Research Alert: At-Home Research Study in People with DM1
Sanguine Biosciences, a provider of at-home clinical research services, is seeking people living with myotonic dystrophy […]
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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 […]
