Protocol for initiation of miglustat treatment in NP-C. Reproduced
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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
Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine
PDF) Goal-oriented therapy with miglustat in Gaucher disease
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
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