Associated Papers
Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis.
Hastings C et al.
Orphanet Journal of Rare Diseases, 2019
Case Report: Ursodeoxycholic acid treatment in Niemann-Pick disease type C; clinical experience in four cases.
Evans WRH et al.
Wellcome Open Research, 2017
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