Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale

Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale

Background: In the 12-month, randomized, double-blind, placebo-controlled Phase 2/3 NPC-002 study (NCT02612129), arimoclomol significantly reduced annual disease progression versus placebo, measured by the 5-domain NPC Clinical Severity Scale (5DNPCCSS). Arimoclomol has been approved in the US for t...

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Main Authors: Eugen Mengel, Marc C. Patterson, Rosalia M. Da Riol, Mireia Del Toro, Federica Deodato, Matthias Gautschi, Stephanie Grunewald, Sabine Weller Grønborg, Paul Harmatz, Julia B. Hennermann, Bénédicte Héron, Esther M. Maier, Agathe Roubertie, Saikat Santra, Anna Tylki-Szymanska, Lisa LaGorio, Elizabeth Berry-Kravis, Forbes D. Porter, Beth Solomon, Louise Himmelstrup, Travis Mickle, Sven Guenther, Christine í Dali
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Arimoclomol
Clinical trial
Niemann-Pick disease type C
NPC Clinical Severity Scale
Validity
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426925000485
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