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ELEVIDYS UPDATE
Breaking News: Sarepta to resume shipments of ELEVIDYS for ambulatory individuals with Duchenne 07/28/25 5:09 PM EDT We are pleased to report that the US FDA has recommended that Sarepta […]
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 the “hot spot” region of exons 45-55, with the goal of submitting applications later this […]
First participant dosed in novel AAV-gene therapy trial for DMD-associated cardiomyopathy
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 prioritization plan. Additionally, Sarepta shared that the U.S. FDA has requested and Sarepta has agreed to include a […]
Craft Your Story
Crafting Your Story for Awareness, Connection, and Change Webinar. Watch HERE
Regulatory Update on Capricor’s Deramiocel for Duchenne
Capricor’s Deramiocel for Duchenne: Capricor Therapeutics, which received early funding from CureDuchenne, has indicated that the FDA has decided that an Advisory Committee meeting is not warranted at this time. […]
CureDuchenne’s latest investment aims to address the limitations of current AAV-delivered gene therapy treatments for Duchenne muscular dystrophy (DMD).
Our recent investment in Entos Pharmaceuticals (https://entospharma.com) aims to explore the potential of their non-viral, redosable Fusogenix PLV platform to deliver full-length dystrophin protein to all muscle groups in a […]
Sarepta pauses Duchenne gene therapy for non-ambulatory individuals after second death due to acute liver failure
Letter to the Community:
REGENXBIO WEBINAR
AFFINITY DUCHENNE®: New Phase 1/2 Interim Functional Data. Watch HERE