Treatment response and neurofilament light chain levels with long-term patisiran in hereditary transthyretin-mediated amyloidosis with polyneuropathy: 24-month results of an open-label extension study.
Simina TicauEmre AldincMichael PolydefkisDavid AdamsTeresa CoelhoMitsuharu UedaCecilia HaleJohn VestPaul Nioinull nullPublished in: Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis (2023)
Long-term patisiran treatment led to sustained improvements in neuropathy and QOL, with NfL demonstrating potential as a biomarker for disease progression and treatment response in ATTRv amyloidosis with polyneuropathy.