Protocol for initiation of miglustat treatment in NP-C. Reproduced
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PDF) Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C
MRx Pipeline October by Magellan Rx Management - Issuu
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study, Orphanet Journal of Rare Diseases
Cyclodextrins applied to the treatment of lysosomal storage disorders - ScienceDirect
Frontiers Therapeutic Strategies For Tay-Sachs Disease
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
