Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial.

Thomas H BrannaganA K WangT CoelhoM Waddington CruzM J PolydefkisP J DyckV Plante-BordeneuveJ L BerkF BarrosoG MerliniI ConceiçãoS G HughesJ KwohS W JungS GuthrieM PollockM D BensonM Gertznull null

Published in: European journal of neurology (2020)

Inotersen slowed disease progression and reduced deterioration of quality of life in patients with hATTR polyneuropathy. Early treatment with inotersen resulted in greater long-term disease stabilization than delayed initiation. Routine platelet and renal safety monitoring were effective; no new safety signals were observed.

Keyphrases

open label
phase iii
clinical trial
phase ii
study protocol
electronic health record
squamous cell carcinoma
data analysis
smoking cessation
replacement therapy
locally advanced