Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...
Ocugen has announced the publication of positive results from its phase 1 GARDian1 trial of OCU410ST, a gene therapy for ...
Study supports favorable safety and tolerability profile and clinically meaningful functional and structural benefits in Stargardt disease ...
Two of Opus's seven gene therapy assets are in clinical trials, with additional programs expected to enter the clinic.
ROCKVILLE, Md., July 10, 2025 /PRNewswire/ -- REGENXBIO Inc. today announced the publication of preclinical results comparing a microdystrophin gene therapy construct that included the C-terminal (CT) ...
A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk independently, his mother said. Edward, from Colchester, has spinal muscular ...
Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...
A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...
AB-1009 program advances to Phase 1/Phase 2; clinical trial initiated in the United StatesTherapy granted United States Food ...
The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source([1]) . The FDA approved three ...
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