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
Changing The Course of NPC: Long-term Evidence for Disease Modification in a Heterogenous Population September 23, 2025
Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to <24 months at study enrollment September 9, 2025
Arimoclomol upregulates expression of genes belonging to the coordinated lysosomal expression and regulation (CLEAR) network
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
Efficacy results across a 12-month double-blind randomized trial and an open-label extension phase of arimoclomol for treatment of Niemann-Pick disease type C in patients treated with miglustat
ADMINISTRATION OF A DUAL-COATED SODIUM PHENYLBUTYRATE (OLPRUVA) SUSPENSION VIA GASTROSTOMY TUBE July 20, 2025
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