PATISIRAN

A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis With a V122I or T60A Mutation

Objective

An observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of hATTR Amyloidosis With a V122I or T60A Mutation

Eligibility

  • Diagnosed with hATTR amyloidosis with polyneuropathy, with a documented V122I or T60A mutation
  • PND score of I-IIIB at the baseline visit
  • Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran