Researchers from The Hospital Santo António and Instituto de Medicina Molecular João Lobo Antunes in Portugal led a team which studied the effects of liver transplant or Vyndaqel (tafamidis), a disease-modifying therapy, on survivability among patients with early-stage familial amyloid polyneuropathy (FAP).

FAP is a rare, slow progressing disease caused by the presence of a mutated blood protein called transthyretin (TTR). This defective protein breaks easily, leading to the accumulation of fibrous deposits on organs and tissues which causes weakness, numbness and pain in the extremities. Treatment options for FAP patients include a liver transplant or taking Vyndaqel (tafamidis) which is designed to delay disease progression for those in the early stages.

As stated by the researchers, the main clinical finding from this survivability study is that Vyndaqel (tafamidis) or liver transplant have “changed dramatically the long-term prognosis for patients with TTR-FAP in the past 25 years, from a progressive, [...] fatal disease to a more chronic condition with treatment leading to delayed disease progression”. In patients younger than 50, Vyndaqel (tafamidis) was shown to reduce the risk of mortality by 91%, while liver transplant lowered the risk by 63% when compared to untreated patients. Learn more by visiting FAP News Today.