Rare disease and gene therapy stocks, battered in recent months by clinical and commercial setbacks, will likely benefit from the sudden departure this past week of Vinayak (Vinay) Prasad, MD, as ...

Wilson Encourages Investors Who Suffered Losses Exceeding $100,000 In Sarepta To Contact Him Directly To Discuss Their ...

Wall Street had a slew of upgrades and downgrades from analysts. Here are some of the major calls for the week: J.P. Morgan ...

I understand the heartbreak of watching someone you love spiral. But compassion cannot mean locking people up,” writes a STAT ...

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

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

In a memo, CEO Stéphane Bancel said the job cuts are needed to align the struggling vaccine maker’s cost structure with the ...

The president overruled his HHS secretary and FDA chief, four people with knowledge of the decision tell POLITICO.

At the 2025 Alzheimer’s Association International Conference, one of the bigger splashes was made by an experimental cardiovascular drug. Newamsterdam Pharma Co. NV presented data showing that its ...

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

Susan Monarez, Ph.D., was confirmed by the Senate Tuesday as director of the Centers for Disease Control and Prevention (CDC) ...

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