Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis.

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作者： Caroline,Hastings ^[1] ; Camilo,Vieira ^[2] ; Benny,Liu ^[3] ; Cyrus,Bascon ^[4] ; Claire,Gao ^[5] ; Raymond Y,Wang ^[6] ; Alicia,Casey ^[7] ; Sharon,Hrynkow ^[8]

作者单位： Department of Pediatric Hematology Oncology, UCSF Benioff Children's Hospital Oakland, 747 52nd Street, Oakland, CA, 94609-1809, USA. caroline.hastings@ucsf.edu. ^[1] Department of Pediatrics, University of California San Francisco, San Francisco, CA, USA. caroline.hastings@ucsf.edu. ^[2] Universidade Federal da Bahia, Clínica Citta, Ed. Mundo Plaza, Av. Tancredo Neves, 620, Sala 1905, Camino dos Árvares, Salvador, Brazil. ^[3] GI & Liver Clinics, Highland Hospital, Alameda Health System, Highland Hospital, Oakland, CA, USA. ^[4] Division of Gastroenterology & Hepatology, Highland Hospital, Alameda Health Systems, Highland Care Pavilion 5th floor, 1411 East 31st Street, Oakland, CA, 94602, USA. ^[5] Department of Pediatric Hematology Oncology, UCSF Benioff Children's Hospital Oakland, 747 52nd Street, Oakland, CA, 94609-1809, USA. ^[6] UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA. ^[7] Present Address: Neuroscience Graduate Program, Brown University, 185 Meeting Street, Box GL-N, Providence, RI, 02912, USA. ^[8] Division of Metabolic Disorders, Children's Hospital of Orange County, CHOC Children's Specialists, 1201 W. La Veta Ave, Orange, CA, 92868, USA. ^[9] Department of Pediatrics, University of California, Irvine School of Medicine, Irvine, CA, 92868, USA. ^[10] Boston Children's Hospital, 300 Longwood Avenue, Boston, MA, 02115, USA. ^[11] CTD Holdings, Inc., P.O. Box 1180, Alachua, FL, 32616, USA. ^[12]

关键词 Hydroxypropyl-beta-cyclodextrin Intravenous administration Investigational new drug, hepatomegaly, splenomegaly, lung disease Niemann-pick disease type C