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

In a striking bipartisan move, the Senate Appropriations Committee voted 26-3 to boost the NIH’s budget by $400 million — rejecting the Trump administration’s proposed $17 billion cut and sweeping ...

The FDA has denied that it plans to combine the Center for Drug Evaluation and Research and Center for Biologics Evaluation ...

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

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 ...

This week on "The Readout LOUD," STAT's biotech reporters parse what Vinay Prasad's ouster means for biotech and the FDA.

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.

Indian-origin scientist Vinay Prasad resigned as the FDA's vaccine and gene therapy chief in just three months after backlash ...