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.

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 ...

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.

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 ...

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.

Congressional Mitochondrial Disease Caucus briefing to explore how FDA drug review processes affect patient care and access for ultra-rare, life-threatening pediatric mitochondrial diseases, using ...

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

The Reverie Cafe, a business offering sugar-free and gluten-free cookies, cakes and bread will close its Central Market location at the end of the month. Owner Charde Barksdale launched The Reverie in ...

NEW YORK – Saol Therapeutics is preparing additional evidence in its effort to convince the US Food and Drug Administration to approve SL1009, a drug it's developing for a rare pediatric mitochondrial ...

FOLSOM, Calif., Dec. 11, 2025 (GLOBE NEWSWIRE) -- The Hope for PDCD Foundation, a parent-led patient advocacy group dedicated to advancing research and treatments for the rare mitochondrial disease ...