Targeted rewriting of the epigenome promises to overcome several challenges in direct gene editing in patient therapies.

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

Prasad, a longtime critic of the FDA’s leniency in drug approvals, joined the agency in May. WASHINGTON — The Food and Drug ...

Fans in need of a superhero fix should head to Marvin Street in Montpelier, where a bright yellow newspaper vending box ...

Two major health agencies are facing leadership reshuffles, with the resignations of the FDA’s Vinay Prasad and NICE’s Dr Sam Roberts.

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to ...

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 U.S. Food and Drug Administration's (FDA) top vaccine official is stepping down after just three months in a role that ...

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 ...