2026

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

February 3, 2026

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

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

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