GeneDx Holdings WGS recently announced a strategic partnership with Komodo Health that is likely to combine its Infinity ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told ...

Scientists at Pacific Northwest Research Institute (PNRI) have overturned a long-held belief in genetics: that inheriting two ...

| Servier is committing to rare neurology for the long term, using focused science and strategic collaborations to bring new ...

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

When our son Peter was diagnosed with an ultra-rare form of muscular dystrophy at the age of 10, the first question we asked was: Is there a treatment? The answer was no. By the time our daughter ...

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...

Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) is one of the best performing pharma stocks in 2025. Amylyx Pharmaceuticals, Inc.

Acanthamoeba keratitis is a rare but blinding parasitic eye disease. Experts share how to avoid it and treat it.