PTC Therapeutics faces pivotal FDA decisions for approvals that could potentially transform their long-term revenues. Click ...

George Tidmarsh has only been at the Center for Drug Evaluation and Research for nine days, but will now add supervision of a ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

A controversial hematologist-oncologist who joined FDA this spring and quickly became its top regulator has left the ...

In the latest episode of STATus Report, Alex Hogan breaks down the confusing, heartbreaking saga of Sarepta and its gene ...

In this week’s edition of InnovationRx, we look at Halle Berry’s menopause startup, Ambience Healthcare’s $243 million ...

Vinay Prasad, who came under fire from patient advocacy groups and Laura Loomer, is gone after less than three months on the ...

Vinay Prasad exits FDA's biologics division following pressure over Sarepta's Elevidys approval and regulatory controversies.

A spokesperson for the Health Department has said that Vinay Prasad did not want to be a "distraction to the great work of the FDA in the Trump administration." ...

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...

The approval to reduce proteinuria for those 12 and older with either of the two severe and rare kidney diseases came on the July 28 PDUFA date, a little more than four years after the company won ...

Sarepta Therapeutics Inc. shares rose sharply early Tuesday after US regulators reversed course and recommended that patients ...