Subgroup Analyses of US EAP Participants With Niemann-Pick Disease Type C Treated With Arimoclomol

August 25, 2026

Real-world use of arimoclomol in Niemann-Pick type C: Findings from a German Compassionate Use Programme

Analysis of NPC1 Genotypes: Findings From the US Arimoclomol Expanded Access Program for Niemann-Pick Disease Type C

April 9, 2026

Long-term safety and effectiveness of arimoclomol adult and pediatricNiemann-Pick disease type C patients in the US early access program (EAP)

February 3, 2026

Real-world safety and effectiveness arimoclomol in patients with NPC:outcomes from the US early access program (EAP) over a 4-year period

Multi-year subgroup analyses of Niemann-Pick disease type C participantstreated with arimoclomol in the US early access program (EAP)

Efficacy of Arimoclomol Combined With Miglustat at Months 3, 6, 9 and 12 of the Double-Blind, Randomized, Placebo-Controlled NPC002 Trial