News

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...
Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts ...
BioMarin's Roctavian gene therapy shows long-term effectiveness in treating severe hemophilia A, reducing bleeding and ...
Using gene therapy to treat hereditary deafness is safe and effective in both children and adults, according to new findings ...
Mouse studies identified the cancer drugs letrozole and irinotecan as a potential combination therapy for Alzheimer's disease ...
The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene ...
RDH12, a gene therapy for RDH12-associated Leber congenital amaurosis (RDH12-LCA) RESEARCH TRIANGLE PARK, N.C., July 23, 2025 ...
The UC Davis Department of Ophthalmology has received a five-year, $3.6 million grant from the National Eye Institute to ...