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The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...
The Purdy family shares their insights on navigating their child’s rare neurodegenerative disease and their direct ...
BioMarin's Roctavian gene therapy shows long-term effectiveness in treating severe hemophilia A, reducing bleeding and ...
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MedPage Today on MSNGene Therapy May Hold Better Outcomes for Congenital Deafness Than Cochlear Implant
Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts ...
15h
News-Medical.Net on MSNBreakthrough gene therapy reverses genetic deafness across age groups
Using gene therapy to treat hereditary deafness is safe and effective in both children and adults, according to new findings ...
RDH12, a gene therapy for RDH12-associated Leber congenital amaurosis (RDH12-LCA) RESEARCH TRIANGLE PARK, N.C., July 23, 2025 ...
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News Medical on MSNUC Davis researchers pioneer safer gene therapy for vision loss
The UC Davis Department of Ophthalmology has received a five-year, $3.6 million grant from the National Eye Institute to ...
The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene ...
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