Persistent Effect Of Arimoclomol In Patients With Niemann-Pick Disease Type C: 24-Month Results From An Open-Label Extension Of A Pivotal Phase 2/3 Study Marc C. Patterson , Eugen Mengel , Rosalia M. Da Riol , Mireia Del Toro , Federica Deodato , Matthias Gautschi , Stephanie Grunewald , Sabine Grønborg , Paul Harmatz , Bénédicte Héron , Esther M. Maier , Agathe Roubertie , Saikat Santra , Anna Tylki-Szymanska , Simon Day , Anne Katrine Andreasen , Marie Aavang Geist , Nikolaj Havnsøe Torp Petersen , Linda Ingemann , Thomas Hansen , Thomas Blaettler , Thomas Kirkegaard , Christine í Dali ANNALS OF NEUROLOGY（2021）
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Neurometabolic Disorders, Rare Diseases
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