Protocol for initiation of miglustat treatment in NP-C. Reproduced
Por um escritor misterioso
Last updated 31 março 2025


Gastrointestinal disturbances and their management in miglustat‐treated patients - Belmatoug - 2011 - Journal of Inherited Metabolic Disease - Wiley Online Library

PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Miglustat in patients with Niemann-Pick disease Type C (NP-C): A

Estimated prevalence of Niemann–Pick type C disease in Quebec

Oxidative stress: a pathogenic mechanism for Niemann-Pick type C disease. - Abstract - Europe PMC

Protocol for initiation of miglustat treatment in NP-C. Reproduced with

Elucidating the mechanism of cyclodextrins in the treatment of Niemann-Pick Disease Type C using crosslinked 2-hydroxypropyl-β-cyclodextrin

NPC1 enables cholesterol mobilization during long‐term potentiation that can be restored in Niemann–Pick disease type C by CYP46A1 activation

Necroptosis in Niemann–Pick disease, type C1: a potential therapeutic target

Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
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