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Academic Journal

Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect

Subjects: Niemann-pick disease type C; Adult-onset; Epidemiology

  • Source: ISSN: 1750-1172 ; Orphanet Journal of Rare Diseases ; https://amu.hal.science/hal-03541909 ; Orphanet Journal of Rare Diseases, 2018, 13 (1), pp.175. ⟨10.1186/s13023-018-0913-4⟩.

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