US regulators laid out new guidelines for approving custom-made treatments for individual patients, a move that could bring ...
SEATTLE — After its world premiere at the 2025 Tribeca Festival. The film, "Matter of Time," had its opening night here in West Seattle. The film is a powerful documentary that blends music, science ...
In FSGS, podocytes are damaged, like a net with torn holes, allowing protein to leak into the urine, creating a harmful ...
The FDA approved the drug for Barth syndrome despite findings by its data reviewers that the treatment, while safe, was no ...
Former tennis star Monica Seles recently revealed she is living with the rare disease myasthenia gravis, which affects 12 in ...
Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is a rare autoimmune condition in which the body's ...
Explore recent changes in rare disease treatments regulation. Discover how these reforms aim to enhance patient access in the UK ...
His family says they have exhausted every option — contacting lawmakers, drug manufacturers, and journalists — and are now ...
PolyActiva and RareSight collaborate to create innovative therapies for rare pediatric retinal diseases, aiming to transform ...
SEPANG: Malaysia will lead efforts for a South-East Asia Declaration and Action Plan on Rare Diseases to improve diagnosis, ...
We have a heartwarming update about a story we brought you just a few weeks ago! Brighton is a three-year-old girl with a rare mitochondrial disease and she is about to step into a whole new way of ...
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