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MDA honors researcher, patient advocate with 2025 Legacy Awards
The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two […]
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Another fall left me feeling like an upside-down turtle
About a month ago, I came home from playing with my church’s music group at […]
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How a Singapore ‘Artist’s Way’ program is rekindling my creativity
Rediscovering my creative self has been one of the most fulfilling yet challenging parts of […]
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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 […]
