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 July 11, 2025
Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network
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 June 17, 2025
Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease April 17, 2025
Efficacy Results from a 12-month Double-blind Randomized Trial of Arimoclomol for the Treatment of Niemann-Pick Disease Type C — Presenting a Rescored 4-domain NPC Clinical Severity Scale February 6, 2025
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 February 3, 2025
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
Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients with Niemann-Pick Type C – Data From 48 Months Open Label Trial
Safety of Arimoclomol in a Pediatric Sub-Study of Niemann-pick Disease Type C Patients Aged 6 To <24 Months at Study Enrollment
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