Researchers suggest that people with a family history of familial amyloid polyneuropathy (FAP) who have psychological problems like distress and paranoia may benefit from…
News
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…
In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to…
At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle…
Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease…
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