Akshay Bhatia won the Arnold Palmer Invitational, crediting his late niece Mia, who he felt was watching over him.

I had my niece definitely watching over me,” Bhatia said of his niece, Mia, who died in December at 6 years old.

Pharma policy watchers were treated Monday to what’s become a regulatory rarity during the second Trump administration — an FDA guidance document requiring notice and comment from the public.

This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite ...

Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare diseases.

The drug was finally approved in September 2025 through the FDA’s accelerated approval pathway, based on improvement in knee extensor muscle strength, for adults and children with Barth syndrome who ...

A research group at the University of Lausanne (Unil) has identified a new mechanism that exposes the vulnerability of tumor ...

Cancer cells are often described as “glutamine addicted,” relying heavily on this amino acid to fuel growth. But new research reveals how some tumors sidestep this vulnerability.