When to decide to enroll a TTR-FAP patient in a Clinical Trial?

Background The Transthyretin-Familial Amyloid Polyneuropathy (TTR-FAP) is a disease caused by deposition of mutant transthyretin (TTR), produced approximately 95% in the liver and the rest in the plexus choroideus and retina. In 1990 the first TTR-FAP patient liver transplant was performed. The liver transplant, which suppresses TTR synthesis, was the only treatment available to modify this disease until 2011, when the European Medicines Agency approved Tafamidis, a TTR stabilizer, for stage I patients (Val30Met and non-Val30Met). To improve upon the pre-existing therapy of liver transplants and given the results of 68% of Tafamidis respondents, other disease-modifying therapeutic approaches were developed: Silencers, Stabilizers, Degraders and Reabsorption agents. In light of these multiple agents clinical trials are the key to improve TTR-FAP treatment.