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
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) November 11, 2024
Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network November 8, 2024
Safety and Efficacy of KP1077 in a Phase 2, Placebo-Controlled, Double-Blind, Randomized Withdrawal Study in Patients with Idiopathic Hypersomnia September 23, 2024
Safety and Efficacy of KP1077 in a Phase 2, Placebo-Controlled, Double-Blind, Randomized Withdrawal Study in Patients with Idiopathic Hypersomnia
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