A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies…
News
A rare mutation of a gene involved in late-onset familial amyloid polyneuropathy occurs at higher rates in one Japanese region, a case study reports. The research,…
Cold and pinprick sensitivity in the hands and electrical impulse speed in nerves can help doctors make an early diagnosis of transthyretin familial amyloid polyneuropathy…
Researchers suggest that people with a family history of familial amyloid polyneuropathy (FAP) who have psychological problems like distress and paranoia may benefit from…
Ionis Pharmaceuticals’ inotersen (IONIS-TTR Rx) continues to demonstrate sustained, long-term benefit for patients with familial amyloid polyneuropathy (FAP), also known as hereditary ATTR…
Ionis Pharmaceuticals and its affiliate Akcea Therapeutics recently announced an exclusive worldwide license to commercialize inotersen and a follow-up therapy, AKCEA-TTR-LRx, formerly…
Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes that cause at least half…
Researchers suggest that guidelines and standardized protocols are needed to evaluate the autonomic nervous system — which regulates certain body processes such as such as…
When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress — SMARD, for short. Hunter…
Researchers have found that imaging contrast agent MIBG can help detect early nerve cell damage affecting the heart before serious tissue damage and cardiac impairment…
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