The FDA approved the drug for Barth syndrome despite findings by its data reviewers that the treatment, while safe, was no ...

Seles’ first symptoms appeared suddenly around three years ago. She began having double vision and weakness in her arms and ...

In FSGS, podocytes are damaged, like a net with torn holes, allowing protein to leak into the urine, creating a harmful ...

Explore recent changes in rare disease treatments regulation. Discover how these reforms aim to enhance patient access in the UK ...

A 15-year-old girl with a rare chromosome disorder received a complete room renovation Friday that her family says will ...

PolyActiva and RareSight collaborate to create innovative therapies for rare pediatric retinal diseases, aiming to transform ...

Early data from the ADAPT JR trial suggest efgartigimod may benefit adolescents with generalized myasthenia gravis, a rare ...

Last month, Jeff Knauss, a Central New York entrepreneur, stepped up in a big way to help those who would not see their SNAP ...

Elafibranor (Iqirvo) was well tolerated and led to sustained improvements in liver enzyme levels and itch severity through 28 weeks in patients with primary sclerosing cholangitis (PSC), according to ...

A biotech stock with strong FDA approval odds this November may see limited upside amid a full valuation and a pending lawsuit.

PAP is a rare lung condition that can be difficult to diagnose because it may mimic many other more common lung diseases,” ...

Around 25 million Americans have a rare genetic disease, and many of them struggle with not only a lack of effective ...