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

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 ...

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 ...

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

Alnylam Pharma raised its revenue outlook as its heart drug sees quick uptake.

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

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

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

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 ...

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 ...