MOESM1 of Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis

Hastings, Caroline; Vieira, Camilo; Liu, Benny; Bascon, Cyrus; Gao, Claire; Wang, Raymond; Casey, Alicia; Hrynkow, Sharon

doi:10.6084/m9.figshare.10022627.v1

13023_2019_1207_MOESM1_ESM.docx (43.7 kB)

MOESM1 of Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis

journal contribution

posted on 2019-10-22, 19:08 authored by Caroline Hastings, Camilo Vieira, Benny Liu, Cyrus Bascon, Claire Gao, Raymond Wang, Alicia Casey, Sharon Hrynkow

Additional file 1. Supplemental Data: Case Summaries on Compassionate Use HPβCD to accompany the manuscript “Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease Type C1: A case report analysis”. Narratives.