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
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
Long-Term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients With Niemann-Pick Disease Type C – Data From a 48-Month Open-Label Trial
Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network
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
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