Treatment with 20 mg a day of oral Vyndaqel (tafamidis) is enough for the therapy to reach the central nervous system (CNS) and eyes…
News
Next-generation sequencing to analyze a panel of 11 specific genes may facilitate the diagnosis of amyloidosis, including familial amyloid polyneuropathy (FAP), according to researchers…
Alnylam Pharmaceuticals’ investigative patisiran treatment improves overall health and prevents worsening of walking ability in patients with familial amyloid polyneuropathy (FAP), according to a…
Familial amyloid polyneuropathy (FAP) patients who do not have a Val30Met mutation exhibit prevalent cardiac and blood vessel dysfunction, but proper blood vessel constriction remains…
Treatment with the investigative therapy Tegsedi (inotersen) provides early and significant improvements in neuropathy symptoms and quality of life in patients with familial amyloid…
The European Commission (EC) has approved Tegsedi (inotersen), by Akcea Therapeutics and Ionis Pharmaceuticals, to treat stage 1 or 2 polyneuropathy in adults with hereditary…
A Phase 3 trial testing patisiran as a therapy for hereditary ATTR amyloidosis with polyneuropathy (also called familial amyloid polyneuropathy, or FAP), showed…
Researchers have reported the case of a Portuguese man with late-onset familial amyloid polyneuropathy (FAP) and a rare combination of heart failure with a…
Treatment with Vyndaqel (tafamidis) or liver transplant can significantly improve the long-term outcome of patients with familial amyloid polyneuropathy (FAP) due to…
A series of potential predictors for identifying patients with familial amyloid polyneuropathy (FAP) and distinguishing it from other diseases with similar symptoms, namely…
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