Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...

Roche Holding AG said on Tuesday it has paused shipments of muscular disorder gene therapy Elevidys in some countries outside ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

During a severe heart attack many heart muscle cells die and are replaced by scar tissue to stabilize the heart wall.

This week on "The Readout LOUD" podcast, a mother whose son has Duchenne muscular dystrophy shares her perspective on Sarepta ...

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 ...

Amid a season of regulatory and scientific advances, experts reveal a culture of data hoarding among cell and gene therapy ...

Shares of Sarepta Therapeutics fell as much as 8% on Tuesday after the company agreed to comply with the U.S. health regulator's request to pause all shipments of its gene therapy Elevidys in the ...

The Swiss company, which markets Elevidys abroad, said it believes the benefit-risk balance to treatment remains positive in ...

The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene ...

The UC Davis Department of Ophthalmology has received a five-year, $3.6 million grant from the National Eye Institute to ...