The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying the agency was investigating the three deaths due to acute liver failure following treatment with Sarepta's AAVrh74 gene therapies.

Using gene therapy to treat hereditary deafness is safe and effective in both children and adults, according to new

The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene therapy for boys with blue cone monochromacy, which impairs color vision and severely affects visual function.

Sarepta Therapeutics said it will temporarily pause all shipments of delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy in the U.S. The company previously refused the FDA's request to halt distribution to all Duchenne patients.

KLOTHO NEUROSCIENCES, INC. - Gene and Cell Therapy of Longevity and Neurodegenerative Diseases. "AAV gene therapy has been proven to be a revolutionary one-time, life-changing treatment, however it is experiencing some crisis caused by high drug price and ...

Shares of Sarepta Therapeutics SRPT nosedived 35.9% on Friday following the death of a patient dosed with one of its experimental gene therapies. The company confirmed that a 51-year-old, non-ambulant (unable to walk) limb-girdle muscular dystrophy (LGMD) patient died due to acute liver failure (ALF).

And participants who received gene therapy also outperformed those who received cochlear implantation in speech-in-noise recognition and music perception, the group noted in JAMA