Protocol for initiation of miglustat treatment in NP-C. Reproduced
Por um escritor misterioso
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MRx Pipeline October by Magellan Rx Management - Issuu
A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1, Lipids in Health and Disease
Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice
Oxidative stress: a pathogenic mechanism for Niemann-Pick type C disease. - Abstract - Europe PMC
Small Molecules: Substrate Inhibitors, Chaperones, Stop-Codon Read Through, and Beyond - Saida Ortolano, 2016
PDF) Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective 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
Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders - ScienceDirect
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Gastrointestinal disturbances and their management in miglustat-treated patients
Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
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