Vyndaqel (tafamidis meglumine) remains safe after more than three years of treatment in patients with familial amyloid polyneuropathy (FAP), with no new safety…
News
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…
Treatment with Tegsedi (inotersen) slows neurological disease progression in people with familial amyloid polyneuropathy (FAP), results from a Phase 2/3 clinical trial show.
Long-term treatment with Onpattro (patisiran) safely and effectively halts or reverses disease progression in people with familial amyloid polyneuropathy (FAP), final data from…
Magnetization transfer ratio (MTR), an MRI scanning tool based on nerve imaging, may help diagnose familial amyloid polyneuropathy (FAP) in…
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and…
Recent Posts
- Scientists ID blood markers that predict heart risk in ATTR-CM
- Navigating amyloidosis treatment can be challenging for older adults
- Tiny skin biopsies may help diagnose transthyretin amyloidosis: New study
- Early diagnosis, treatment improves survival in ATTR-CM: Study
- FDA lifts clinical hold on Phase 3 trial of nex-z for hATTR-PN