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Efficacy and safety of arimoclomol in Niemann‐Pick disease ..:
Mengel, Eugen
;
Patterson, Marc C
;
Da Riol, Rosalia M
...
qt2zk0w272. , 2021
Link:
https://escholarship.org/uc/item/2zk0w272
RT Journal T1
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
UL https://suche.suub.uni-bremen.de/peid=base-ftcdlib:oai:escholarship.org:ark:_13030_qt2zk0w272&Exemplar=1&LAN=DE A1 Mengel, Eugen A1 Patterson, Marc C A1 Da Riol, Rosalia M A1 Del Toro, Mireia A1 Deodato, Federica A1 Gautschi, Matthias A1 Grunewald, Stephanie A1 Grønborg, Sabine A1 Harmatz, Paul A1 Héron, Bénédicte A1 Maier, Esther M A1 Roubertie, Agathe A1 Santra, Saikat A1 Tylki‐Szymanska, Anna A1 Day, Simon A1 Andreasen, Anne Katrine A1 Geist, Marie Aavang A1 Petersen, Nikolaj Havnsøe Torp A1 Ingemann, Linda A1 Hansen, Thomas A1 Blaettler, Thomas A1 Kirkegaard, Thomas A1 Dali, Christine PB eScholarship, University of California YR 2021 K1 Clinical Research K1 Clinical Trials and Supportive Activities K1 6.1 Pharmaceuticals K1 Evaluation of treatments and therapeutic interventions K1 Adolescent K1 Child K1 Preschool K1 Disease Progression K1 Double-Blind Method K1 Female K1 Humans K1 Hydroxylamines K1 Internationality K1 Male K1 Niemann-Pick Disease K1 Type C K1 Prospective Studies K1 Severity of Illness Index K1 Treatment Outcome K1 Young Adult K1 arimoclomol K1 biomarker K1 double-blindplacebo-controlled K1 heat shock protein K1 Niemann-Pick disease type C K1 NPC clinical severity scale K1 Clinical Sciences K1 Genetics & Heredity JF qt2zk0w272 LK http://dx.doi.org/https://escholarship.org/uc/item/2zk0w272 DO https://escholarship.org/uc/item/2zk0w272 SF ELIB - SuUB Bremen
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