The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease cerebral folate deficiency (CFD) in both children and adults, the same ...

Acadia Pharmaceuticals said that drug regulators in Europe are leaning toward rejecting its application for a neurodevelopmental disorder treatment. The company said Monday it was informed by a ...

John William Klinefelter, aged 83, of Great Falls, Montana, passed away on January 13, 2026, after a courageous battle with pancreatic cancer. He was surrounded by family at Peace Hospice, where he ...

Credit: Acadia. Daybue Stix, a strawberry-flavored trofinetide powder mixable with water-based liquids, gains FDA approval for Rett syndrome treatment. Acadia expects to have the new formulation ...

Biologics, including GLP-1 analogs and PCSK9 inhibitors, are advancing treatment options for metabolic syndrome components like type 2 diabetes and dyslipidemia. GLP-1 receptor agonists improve ...

Klinefelter syndrome can cause problems with learning and sexual development in guys. It's a genetic condition (meaning a person is born with it). Klinefelter syndrome only affects males. It happens ...

According to reporting from Reuters, reviewers at the agency pointed to an inability to differentiate from placebo to justify rejecting the drug, but an FDA office director approved the drug anyway.

“The most important lifestyle factor [in FCS] is a strict low-fat diet,” says David H. Hsi, MD, chief of cardiology and co-director of the Heart and Vascular Institute at Stamford Health in ...

Walker Burger always loved baseball, but attending the games of his favorite team, the Atlanta Braves, used to be a physical challenge. In fact, every form of exercise was difficult. Burger, 35, has a ...

The FDA has granted accelerated approval for the elamipretide injection, the first treatment for Barth syndrome in patients weighing at least 30 kg. Barth syndrome is a life threatening disease of the ...

The FDA on Friday granted accelerated approval to elamipretide (Forzinity) as the first treatment for the ultra-rare Barth syndrome, which affects approximately 150 Americans. Elamipretide once-daily ...

After a tumultuous journey filled with delays and rejection, the FDA has approved Stealth BioTherapeutics’ treatment for the ultrarare disease Barth syndrome, marking the peptide as the first therapy ...