The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...

An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...

PTC Therapeutics faces pivotal FDA decisions for approvals that could potentially transform their long-term revenues. Click ...

The FDA greenlit multiple new drugs this month and issued some notable label expansions, including for Eli Lilly’s Kisunla.

Donald Trump reportedly ignored calls from Health Secretary Robert F. Kennedy Jr. not to fire the top vaccine official at the Food and Drug Administration after a pressure campaign from far-right ...

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

After our editorial, the agency relents to allow a Duchenne treatment.

The FDA investigated the death of a boy in Brazil on Elevidys and concluded it was unrelated to the treatment.