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MedPage Today2d
Drugmaker Refuses FDA's Request to Pull Gene Therapy Tied to Patient Deaths
The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...
Neurology Advisor2h
Child’s Rare Neurodegenerative Disease Fuels Therapy Innovation: Paxton’s Story
The Purdy family shares their insights on navigating their child’s rare neurodegenerative disease and their direct ...
The Manila Times6h
Opus Genetics and the Global RDH12 Alliance Partner to Advance RDH12 Gene Therapy for Inherited Childhood Blindness
RDH12, a gene therapy for RDH12-associated Leber congenital amaurosis (RDH12-LCA) RESEARCH TRIANGLE PARK, N.C., July 23, 2025 ...
MedPage Today on MSN1d
Gene 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 ...
Managed Healthcare Executive16h
Roctavian Gene Therapy Controls Bleeding for Five Years in Severe Hemophilia A
BioMarin's Roctavian gene therapy shows long-term effectiveness in treating severe hemophilia A, reducing bleeding and ...
News-Medical.Net on MSN15h
Breakthrough 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 ...
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News Medical on MSN15h
UC 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 ...
Managed Healthcare Executive1d
Gene Therapy for Rare Eye Disease to Advance to Human Trial
The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene ...