Positive real-world effectiveness of tafamidis for delaying disease progression in transthyretin familial amyloid polyneuropathy
نویسندگان
چکیده
Background Tafamidis (Vyndaqel) was approved by the EMA in 2011 and is emerging as the standard of care for transthyretin familial amyloid polyneuropathy (TTR-FAP) in clinical settings. Efficacy was demonstrated in the clinical trials, yet little is known about its real-world effectiveness. A global disease registry, the Transthyretin Amyloidosis Outcomes Survey (THAOS), collects data on both treated and untreated patients from real-world settings. Ethics committee approval was obtained prior to patient enrolment.
منابع مشابه
Early intervention with tafamidis provides long-term benefit in delaying neurological progression in patients with transthyretin familial amyloid polyneuropathy
Background Tafamidis is a transthyretin (TTR) stabilizer approved to delay neurological progression associated with stage 1 TTR familial amyloid polyneuropathy (FAP). A placebocontrolled, randomized 18-month registration trial allowed for continued evaluation of patients receiving tafamidis (20 mg oral once-daily) through an ongoing open label extension study. The effectiveness of tafamidis for...
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Background Tafamidis is a transthyretin (TTR) stabilizer able to prevent mutated TTR tetramer dissociation into amyloidogenic monomers. There have been a few encouraging studies on safety and long-term efficacy of Tafamidis in early-onset Val30Met TTR-familial amyloid polyneuropathy (TTR-FAP) patients. However, less is known about its efficacy in later stages of the disease and in non-Val30Met ...
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