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ELEVIDYS Update
PPMD acknowledges the media reports that the U.S. Food and Drug Administration (FDA) will request […]
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Sarepta Therapeutics Announces Update on ELEVIDYS Label, Company Restructuring
Yesterday afternoon, Sarepta issued a press release and community letter which included updates related to […]
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Dosing begins in gene therapy trial for DMD-linked heart disease
The first patient has been dosed as part of a Phase 1b clinical trial of […]
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New preclinical data from Precision BioSciences’s gene editing program for Duchenne
Precision BioSciences is developing a novel gene editing approach for individuals with dystrophin mutations in […]
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With FSHD, dealing with pain is a daily challenge for me
In a daily devotional I’m reading titled “Incurable Faith: 120 Devotions of Lasting Hope for […]
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First participant dosed in novel AAV-gene therapy trial for DMD-associated cardiomyopathy
Medera, and its clinical division Sardocor, announced that the first participant was dosed with AAV1.SERCA2a […]
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Important Update: Sarepta Therapeutics Announces Strategic Restructuring and Pipeline Prioritization Plan to Maintain Long-term, Sustainable Growth and Provides Update on ELEVIDYS Label
CureDuchenne is sharing a community letter from Sarepta announcing a strategic company restructuring and pipeline […]
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MDA Ambassador Guest Blog: The Joy of Accessible Sailing
Mike Huddleston is 62 years old. He is originally from California and now lives in […]
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Capricor plans for deramiocel resubmission after FDA rejection
Capricor Therapeutics will resubmit its application for U.S. Food and Drug Administration (FDA) approval of […]
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Finding solace: Why I need July’s slower pace
July is my favorite month. Many people who know me might be surprised by that, […]
