A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy, did not achieve its primary endpoint, according to findings ...
Opens in a new tab or window SAVANNAH, Ga. -- Delandistrogene moxeparvovec (Elevidys), a gene therapy approved to treat Duchenne muscular dystrophy, missed its primary endpoint but differences ...
Sarepta's gene therapy – like rivals from Pfizer and Solid Biosciences – codes for a shortened form of the dystrophin protein that is deficient in patients with the muscle-wasting disease.
We identified a novel and significant association between Duchenne muscular dystrophy and autism spectrum ... a specific region within the dystrophin gene that is altered, creating either a ...
New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. Updated ...
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FDA Expands Sarepta's (SRPT) DMD Gene Therapy LabelShares of Sarepta SRPT rose nearly 34% in after-market trading on Thursday after it announced that the FDA approved the expanded use of its Duchenne muscular dystrophy (DMD) gene therapy Elevidys.
the controversial Duchenne muscular dystrophy gene therapy that Roche markets outside of the U.S. through a licensing deal with Sarepta. With phase 3 miss, Pfizer's DMD candidate is no threat to ...
New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...
More information: Jerry R. Mendell et al, AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial, Nature Medicine (2024). DOI: 10.1038/s41591-024-03304-z ...
Confounding data from a Pfizer clinical trial has rattled the field of gene therapy for Duchenne muscular dystrophy, raising more questions about the regulatory standard used to approve a ...
New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...
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