CRX-1008, an investigational therapy also known as SOM0226 or tolcapone, is safe and well-tolerated by patients as a treatment for familial amyloid polyneuropathy (FAP),…
News
The characteristic toxic buildup of mutant transthyretin (TTR) protein clumps in familial amyloid polyneuropathy (FAP) affects the development of nerve cell fibers by…
Alnylam Pharmaceuticals announced a potential new biannual dosing regimen for vutrisiran, its investigational treatment for familial amyloid polyneuropathy (FAP) and other forms…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for…
Vyndaqel (tafamidis meglumine) remains safe after more than three years of treatment in patients with familial amyloid polyneuropathy (FAP), with no new safety…
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for…
Using ultrasound to measure the size of the median nerve — which runs down the upper arm and continues into the forearm and hand…
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal…
Treatment with Tegsedi (inotersen) eased a range of neuropathy symptoms in people with familial amyloid polyneuropathy (FAP), new data from the NEURO-TTR Phase 2/3 trial…
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find…
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