文献
J-GLOBAL ID:201802238160120437
整理番号:18A1154915
リソソーム酸性リパーゼ欠損症に対するセベリパーゼαで治療した乳児における生存: オープンラベル,多施設,用量漸増試験【JST・京大機械翻訳】
Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study
著者 (18件):
Jones Simon A.
(Manchester Centre for Genomic Medicine, 6th floor, St Mary’s Hospital, Central Manchester Foundation Trust, University of Manchester, Manchester, UK)
,
Rojas-Caro Sandra
(Synageva BioPharma Corp., Lexington, USA)
,
Quinn Anthony G.
(Synageva BioPharma Corp., Lexington, USA)
,
Quinn Anthony G.
(Present: IDBioPharm Consulting, LLC, Boston, USA)
,
Friedman Mark
(Alexion Pharmaceuticals, Inc., New Haven, USA)
,
Marulkar Sachin
(Alexion Pharmaceuticals, Inc., New Haven, USA)
,
Ezgu Fatih
(Gazi University Faculty of Medicine, Gazi Hospital, Besevler Ankara, Turkey)
,
Zaki Osama
(Ain Shams University Pediatrics Hospital, Heliopolis, Cairo, Egypt)
,
Gargus J. Jay
(University of California, Irvine, Irvine, USA)
,
Hughes Joanne
(Temple Street Children’s University Hospital, Dublin, Ireland)
,
Plantaz Dominique
(Hopital Couple-Enfant CHU Grenoble, Grenoble, France)
,
Vara Roshni
(Evelina Children’s Hospital, London, UK)
,
Eckert Stephen
(Synageva BioPharma Corp., Lexington, USA)
,
Arnoux Jean-Baptiste
(Hopital Necker-Enfants Malades and IMAGINE Institute, Paris, France)
,
Brassier Anais
(Hopital Necker-Enfants Malades and IMAGINE Institute, Paris, France)
,
Sang Kim-Hanh Le Quan
(Hopital Necker-Enfants Malades and IMAGINE Institute, Paris, France)
,
Valayannopoulos Vassili
(Hopital Necker-Enfants Malades and IMAGINE Institute, Paris, France)
,
Valayannopoulos Vassili
(Present: Sanofi Genzyme, Cambridge, USA)
資料名:
Orphanet Journal of Rare Diseases (Web)
(Orphanet Journal of Rare Diseases (Web))
巻:
12
号:
1
ページ:
25
発行年:
2017年
JST資料番号:
U7532A
ISSN:
1750-1172
資料種別:
逐次刊行物 (A)
記事区分:
原著論文
発行国:
イギリス (GBR)
言語:
英語 (EN)