Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial July 16, 2025
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
Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study
Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment March 1, 2023
Patterns of abuse and routes of administration for immediate-release hydrocodone combination products January 26, 2023
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