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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 ...
Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts ...
Using 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 ...
The UC Davis Department of Ophthalmology has received a five-year, $3.6 million grant from the National Eye Institute to ...
Sarepta Therapeutics said it will temporarily pause all shipments of delandistrogene moxeparvovec (Elevidys) for Duchenne ...