Protocol for initiation of miglustat treatment in NP-C. Reproduced
Por um escritor misterioso
Last updated 16 setembro 2024
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Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
MRx Pipeline October by Magellan Rx Management - Issuu
Phenotypic expression of swallowing function in Niemann–Pick disease type C1, Orphanet Journal of Rare Diseases
Miglustat - wikidoc
Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrin. - Abstract - Europe PMC
Elucidating the mechanism of cyclodextrins in the treatment of Niemann-Pick Disease Type C using crosslinked 2-hydroxypropyl-β-cyclodextrin
PDF) Goal-oriented therapy with miglustat in Gaucher disease
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)
Distinct Niemann-Pick Disease Type C Clinical, Cytological, and Biochemical Phenotype in an Adult Patient With 1 Mutated, Overexpressed NPC1 Allele - Julia Jecel, Klaus Harzer, Eduard Paschke, Stefanie Beck-Wödl, Peter Bauer, Milos
Oxidative stress: a pathogenic mechanism for Niemann-Pick type C disease. - Abstract - Europe PMC
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