A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1

INTRODUCTION: Niemann-Pick C (NPC) is an autosomal recessive disease due to defective NPC1 or NPC2 proteins resulting in endo-lysosomal storage of unesterified cholesterol in the central nervous system and liver. Acute liver disease in the newborn period may be self-limited or fatal. 2-hydroxypropyl...

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Detalles Bibliográficos
Autores principales: Reynolds, Margaret, Linneman, Laura A., Luna, Sofia, Warner, Barbara B., Turmelle, Yumirle P., Kulkarni, Sakil S., Jiang, Xuntian, Khanna, Geetika, Shinawi, Marwan, Porter, Forbes D., Ory, Daniel S., Cole, F. Sessions, Dickson, Patricia I.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8170172/
https://www.ncbi.nlm.nih.gov/pubmed/34113546
http://dx.doi.org/10.1016/j.ymgmr.2021.100772

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