Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk.

Investors showed growing mistrust of the Sarepta team, repeatedly asking why the Cambridge company had not disclosed the ...

US Food and Drug Administration Commissioner Marty Makary said he’s “taking a hard look” at whether a gene therapy from ...

The U.S. Food and Drug Administration will request Sarepta Therapeutics to voluntarily stop all shipments of its gene therapy ...

UC Davis MIND Institute researchers Randi and Paul Hagerman are calling for increased awareness and screening for fragile ...

Rocket Pharmaceuticals RCKT announced that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation ...

The death will likely further stoke safety concerns around Sarepta's gene therapies, as well as raise questions around the ...

A third patient has died from acute liver failure after receiving a gene therapy treatment from Sarepta Therapeutics, the biotech company confirmed to Bloomberg on July 18. The patient died while ...

Thirty-three states, the District of Columbia, and Puerto Rico will participate in CMS’ voluntary Cell and Gene Therapy (CGT) ...

According to multiple reports, a 51-year-old man with limb-girdle muscular dystrophy died from acute liver failure after ...

The Purdy family shares their insights on navigating their child’s rare neurodegenerative disease and their direct ...

Discover how Genome Valley is emerging as an essential partner in global biopharma and how it is now venturing into cell and gene therapy.