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 ...

What has Wall Street been buzzing about this week? Here are the top 5 Buy calls and the top 5 Sell calls made by Wall Street’s best analysts ...

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 ...

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.

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.