The effect of the familial amyloid polyneuropathy (FAP) hallmark protein transthyretin (TTR) on cells is associated with its specific three-dimensional shape, according to Italian researchers. Their study, “A FTIR microspectroscopy study of the structural and biochemical perturbations induced by natively folded and aggregated transthyretin in HL-1…
Treatment with the investigative therapy Tegsedi (inotersen) provides early and significant improvements in neuropathy symptoms and quality of life in patients with familial amyloid polyneuropathy (FAP), according to results from a pivotal Phase 2/3 clinical trial. These results, announced by Ionis Pharmaceuticals, the treatment’s developer, and its affiliate, Akcea…
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…
Advancing the study of rare diseases, including familial amyloid polyneuropathy (FAP), is the goal of a new research institute to be founded at the University of Massachusetts (UMass) Medical School. The new institute is possible thanks to a substantial donation from the Li Weibo Charitable Foundation in China. Li Weibo, the…
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