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

Perseverance Is Key for Regulatory Success in Ultra-Rare Diseases – Key Learnings from Arimoclomol’s Regulatory Journey

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

Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network

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