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Boys with DMD given Elevidys two years ago still showing motor gains
Two years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl), motor function continues to improve
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Sarepta’s EMBARK study data shows continuing benefits to ambulatory individuals treated with their microdystrophin gene therapy, Elevidys
Sarepta Therapeutics shared positive topline results from Part 2 of the EMBARK study, showing that
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Sarepta Therapeutics Announces Results from Part 2 of EMBARK Study of ELEVIDYS
Sarepta Therapeutics, Inc. has announced positive topline results from Part 2 of EMBARK, the company’s
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Update from PPMD’s 2025 Duchenne Healthcare Professionals Summit
PPMD recently convened over 360 healthcare professionals, researchers, and industry partners, including representatives from the
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FDA awards fast track designation to DM1 treatment DYNE-101
The U.S. Food and Drug Administration (FDA) has granted fast track designation to DYNE-101, a
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What success looks like for me as a DMD parent
Parenting is the most challenging job I’ve ever had. When my role as a parent
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Avidity plans to submit for FDA Accelerated Approval for Duchenne exon 44 skipping at the end of 2025; Open-label extension trial is still recruiting
Amenable to skipping exon 44 Avidity Biosciences, which received early funding from CureDuchenne, plans to
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The CureDuchenne International Outreach Program: Expanding Duchenne Care Beyond Borders
By Debra Miller, Founder and CEO of CureDuchenne Duchenne International Outreach: Bridging Gaps in Duchenne
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Corticosteroid at low dose may help with muscle damage, inflammation
Once a week treatment with a corticosteroid was seen to lower some biomarkers of muscle
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Race to End Duchenne Kicks Off 20th Anniversary at Walt Disney World Marathon Weekend
The Race to End Duchenne program is celebrating our 20th anniversary in 2025, and we
