PRX004, an investigational treatment by Prothena, can reduce the progression of nerve damage (neuropathy) and improve heart function in people…
Articles by Marisa Horak, MS
Onpattro (patisiran) results in sustained improvements in polyneuropathy, quality of life, and other symptoms among people with familial…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
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…