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 ...

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...

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

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

Vinay Prasad’s ouster as the Food and Drug Administration’s top vaccine and gene therapy regulator came after frustration ...

Niemann-Pick Disease Type C (NPC) is a rare genetic disorder caused by mutations in the NPC1 or NPC2 genes, leading to harmful lipid accumulation in organs like the liver and brain. Symptoms, often ...

A biopharmaceutical research company with ties in Andover has laid off a third of its workforce as one of the industry leaders in gene therapy goes through a “strategic restructuring.” Sarepta ...

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 ...