arimoclomol Miplyffa NPC
Selected indexed studies
- 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]
- 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. (Mol Genet Metab Rep, 2025) [PMID:40520915]
_Worker-drafted node — pending editorial review._
Connections
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Sources
- 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
- Alterations in Proteostasis Mechanisms in Niemann-Pick Type C Disease. (2024) pubmed
- Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease. (2025) pubmed
- Levacetylleucine (N-acetyl-l-leucine) for Niemann-Pick disease type C. (2025) pubmed
- Treatment trials in Niemann-Pick type C disease. (2021) pubmed
- FDA approval of Miplyffa and Aqneursa: A dual breakthrough for the treatment of Neimann-Pick disease type C. (2025) pubmed