miglustat NPC
Selected indexed studies
- Niemann-Pick disease type C. (Orphanet J Rare Dis, 2010) [PMID:20525256]
- Arimoclomol: First Approval. (Drugs, 2025) [PMID:39715913]
- 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. (J Inherit Metab Dis, 2021) [PMID:34418116]
_Worker-drafted node — pending editorial review._
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Sources
- Miglustat in Niemann-Pick disease type C patients: a review. (2018) pubmed
- Arimoclomol: First Approval. (2025) pubmed
- 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. (2021) pubmed
- 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. (2025) pubmed
- 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. (2025) pubmed
- Niemann-Pick Disease Type C. (1993) pubmed
- Niemann-Pick disease type C. (2010) pubmed
- Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry. (2020) pubmed
- Miglustat: a review of its use in Niemann-Pick disease type C. (2014) pubmed
- Evaluation of the safety and efficacy of miglustat for the treatment of Chinese patients with Niemann-Pick disease type C: A prospective, open-label, single-arm, phase IV trial. (2024) pubmed