Vyndaqel (tafamidis) significantly delays the progression of familial amyloid polyneuropathy (FAP) and improves the quality of life of FAP patients, a real-world study shows. These results support data from clinical trials showing that Vyndaqel is an effective treatment for FAP. The study, “Positive Effectiveness of Tafamidis…
Studying the interaction between different amyloid (abnormal disease-causing) proteins may provide insight into the treatment of diseases such as familial amyloid polyneuropathy (FAP), an Indian study suggests. The study, “Q-Rich Yeast Prion [PSI+] Accelerates Aggregation of Transthyretin, a Non-Q-Rich Human Protein,” appeared in the journal Frontiers in…
Akcea Therapeutics has completed a deal giving it the global marketing rights to its affiliate Ionis Pharmaceuticals’ familial amyloid polyneuropathy therapy inotersen. The deal also gives Akcea rights to a follow-up treatment known as AKCEA-TTR-L Rx, formerly known as IONIS-TTR-L Rx. Inotersen was designed for hereditary forms of FAP, which…
Familial amyloid polyneuropathy (FAP) is linked to important alterations of the autonomic nervous system, which regulates certain body processes such as breathing, heart rate, and digestion, according to researchers. This finding resulted from a study led by Alejandra Gonzalez‑Duarte, a neurologist at the Salvador Zubirán National Institute of Medical…
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 for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…
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, “Two cases of late onset familial amyloid polyneuropathy with a Glu61Lys transthyretin variant,” was published in the Journal of the Neurological Sciences. Most…
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 (TTR-FAP), a study reports. The research, “Transthyretin familial amyloid polyneuropathy (TTR-FAP): Parameters for early diagnosis,” appeared in the journal Brain & Behavior. Transthyretin…
Researchers suggest that people with a family history of familial amyloid polyneuropathy (FAP) who have psychological problems like distress and paranoia may benefit from psychological support before diagnostic testing. The study, “Long-term predictors for psychological outcome of pre-symptomatic testing for late- onset neurological diseases,” was published in the…
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 (hATTR) amyloidosis. Recent positive results of a study were the subject of several presentations at the 16th International Symposium on Amyloidosis (ISA) being held March…
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 known as IONIS-TTR-LRx. Inotersen is a therapy candidate being evaluated for the treatment of familial amyloid polyneuropathy (FAP). In May 2017, Ionis announced positive results from…