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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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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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Your Voice Is Everything
When the Duchenne and Becker community speaks, the world listens — not just because of […]
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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 […]
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Del-zota for DMD awarded FDA breakthrough therapy designation
The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to delpacibart zotadirsen, known […]
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Avidity Biosciences granted Breakthrough Therapy Designation for exon-44 skipping therapeutic, del-zota
CureDuchenne was an early investor in Avidity Biosciences in 2016, and congratulates the company for receiving Breakthrough Therapy […]
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How art can foster connection in the disability community
Last Sunday, my girlfriend, Amanda, and I co-led an art workshop that brought us both […]
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FDA Grants Breakthrough Therapy Designation to Avidity’s del-zota
Avidity Biosciences, Inc. today shared that the U.S. Food and Drug Administration (FDA) has granted […]
