The progressive nature of hereditary transthyretin amyloidosis polyneuropathy (hATTR-PN), also known as familial amyloid polyneuropathy (FAP), limits patients’ health-related quality of life across all measures, with pain a particular problem, and is linked to depression or anxiety in carriers, a study from Portugal found. Earlier diagnosis and treatment may…
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…
Shire, Microsoft and Rare Diseases Europe (EURORDIS)Â have established a partnership to end the diagnostic challenge endured by patients with rare diseases like familial amyloid polyneuropathy (FAP). According to the newly established Global Commission to End the Diagnostic Odyssey for Children, it takes an average of…