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Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C

Subjects: Male; Pediatrics; Vesicular Transport Proteins

  • Source: Orphanet Journal of Rare DiseasesOrphanet Journal of Rare Diseases, BioMed Central, 2012, 7 (1), pp.36. ⟨10.1186/1750-1172-7-36⟩Orphanet Journal of Rare Diseases, 2012, 7 (1), pp.36.

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