FDA accepts setmelanotide sNDA (PDUFA 12/20/2025), but revenue outlook is tepid; watch Prader-Willi catalyst—read now.

Maria Baum was born with Williams syndrome, a genetic neurodevelopmental disorder strongly linked to cardiovascular disease ...

A Beaverton family that has spent years searching for a replacement gene for their son’s rare genetic disorder says they are ...

Yentli Soto Albrecht, who is a genetic carrier of ALS, is working on research at Penn that could one day prove life-saving.

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

Four years later, the Phams are searching for a cure after their son was diagnosed with a rare neurological disorder, hoping to raise funds for clinical trials.

Researchers at VIB and Antwerp University have identified a major genetic risk factor for a rare form of frontotemporal dementia. The discovery, published today in Nature Genetics, provides a ...

Meet the luckiest people on Earth — individuals carrying a rare genetic mutation that prevents wrinkles from forming even as they age well into their 80s. These genetic lottery winners have provided ...

The prescription drug leucovorin is getting a label update, but it's not what that the US Food and Drug Administration suggested during a White House briefing in September.

Sildenafil—an active ingredient also marketed under the name of Viagra—improves symptoms in patients with Leigh syndrome.