Arimoclomol for the Treatment of Niemann-Pick Disease Type C in a Real-World Setting: Long-Term Outcomes From an Expanded Access Program in the United States

Long-Term Effectiveness and Safety Evaluation of Arimoclomol Treatment in Patients With Niemann-Pick Disease Type C – Data From the Pivotal Study and Open-Label Extension

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