GeneDx, which specializes in diagnostics for rare and ultra-rare genetic disorders, is helping to get kids the diagnostic attention they need as part of Diagnosis is Power. Currently, one in 10 ...
There’s a good chance that you haven’t heard about a rare condition called chromhidrosis. In a nutshell, the person afflicted with the disease produces colored sweat, from yellow to green, even red ...
PAP is a rare lung condition that can be difficult to diagnose because it may mimic many other more common lung diseases,” ...
New clinical-stage company will accelerate treatments for patients with ultra-rare diseases worldwide PHILADELPHIA, Oct. 7, 2025 /PRNewswire/ -- Gemma Biotherapeutics ("GEMMABio") – a leading global ...
Hosted on MSN
Meet your worm avatar: How microscopic worms are helping find new drugs for rare diseases
New research from the MRC Laboratory of Medical Sciences (LMS) provides a powerful, scalable method for finding treatments for rare genetic diseases using tiny, transparent worms. Subscribe to our ...
The FDA has outlined a new review process for drugs and biologics designed to treat ultrarare genetic diseases that would allow a single-arm trial, plus other supportive data, to serve as pivotal ...
The drug, discovered by Japan Tobacco and once in the hands of Merck, was licensed by BridgeBio in 2019.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback