Published in
Wiley, Journal of Inherited Metabolic Disease, 6(44), p. 1463-1480, 2021
DOI: 10.1002/jimd.12428
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Efficacy and safety of arimoclomol in Niemann‐Pick disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatment
Journal article published in 2021 by
Eugen Mengel 
,
Marc C. Patterson,
Christine í. Dali,
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
and other authors.
,
Marc C. Patterson,
Christine í. Dali,
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
and other authors.
This paper is made freely available by the publisher.
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