A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Articles by Marisa Wexler, MS
CRX-1008, an investigational therapy also known as SOM0226 or tolcapone, is safe and well-tolerated by patients as a treatment for…
Using ultrasound to measure the size of the median nerve — which runs down the upper arm and continues…
Treatment with Tegsedi (inotersen) eased a range of neuropathy symptoms in people with familial amyloid polyneuropathy (FAP), new data from…
Treatment with Tegsedi (inotersen) slows neurological disease progression in people with familial amyloid polyneuropathy (FAP), results from a…
Levels of calprotectin, a marker of inflammation, are elevated in the feces of people with familial amyloid polyneuropathy…
The Italian Medicines Agency has added Tegsedi (inotersen), a treatment for familial amyloid polyneuropathy (FAP), to the…
Treatment with Onpattro (patisiran) improves multiple aspects of quality of life in people with familial amyloid polyneuropathy (FAP),…
A type of surgery that involves removal of the eye’s “gel” can improve vision in people with familial amyloid…
Long-term treatment with Vyndaqel (tafamidis) may reduce the risk of death in people with familial amyloid polyneuropathy…