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Efficacy Results from A 12-month Double-blind Randomised Trial of Arimoclomol for Treatment of Niemann-Pick Disease Type C – Presenting A Rescored 4-domain NPC Clinical Severity Scale

Qualitative Assessment of the Validity and Standardization of the Swallow Domain in the 5-domain Niemann-Pick Disease Type C (NPC) Clinical Severity Scale (5DNPCCSS) And Analysis in an NPC Clinical Trial Data Set

Arimoclomol Safety Profile in the Treatment of Niemann-pick Disease Type C In a Real-world Setting: Long-term Safety Data from an Expanded Access Program in the United States

Qualitative Assessment of the Validity and Standardization of the Swallow Domain in the 5-Domain Niemann-Pick disease type C (NPC) Clinical Severity Scale (5DNPCCSS)