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The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...
RDH12, a gene therapy for RDH12-associated Leber congenital amaurosis (RDH12-LCA) RESEARCH TRIANGLE PARK, N.C., July 23, 2025 ...
Fairy disease can shorten lifespans and damage organs. A new treatment offers sufferers of the disorder a reprieve from ...
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MedPage Today on MSNGene Therapy May Hold Better Outcomes for Congenital Deafness Than Cochlear ImplantYouth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts ...
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News-Medical.Net on MSNBreakthrough gene therapy reverses genetic deafness across age groupsUsing gene therapy to treat hereditary deafness is safe and effective in both children and adults, according to new findings ...
BioMarin's Roctavian gene therapy shows long-term effectiveness in treating severe hemophilia A, reducing bleeding and ...
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News Medical on MSNUC Davis researchers pioneer safer gene therapy for vision lossThe UC Davis Department of Ophthalmology has received a five-year, $3.6 million grant from the National Eye Institute to ...
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MedPage Today on MSNCancer Drugs for Alzheimer's? Gene Therapy Distribution Halted; Reframing APOE4Sarepta Therapeutics said it will temporarily pause all shipments of delandistrogene moxeparvovec (Elevidys) for Duchenne ...
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