Phase 2 open-label study with a placebo-controlled drug withdrawal period of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with Alagille Syndrome: 48-week interim efficacy analysis

被引：8

作者：

Gonzales, Emmanuel ^{[1
]}

Sturm, Ekkehard ^{[2
]}

Stormon, Michael ^{[3
]}

Sokal, Etienne ^{[4
]}

Hardikar, Winita ^{[5
]}

Lacaille, Florence ^{[6
]}

Gliwicz, Dorota ^{[7
]}

Hierro, Loreto ^{[8
]}

Jaecklin, Thomas ^{[9
]}

Gu, Joan ^{[10
]}

Desai, Nirav K. ^{[11
]}

Dorenbaum, Alejandro ^{[12
]}

Kennedy, Ciara ^{[13
]}

Baker, Alastair ^{[14
]}

Jacquemin, Emmanuel ^{[1
]}

机构：

[1] Univ Paris Sud Paris Saclay, Hop Bicetre, AP HP, Hepatol Pediat, Paris, France

[2] Univ Hosp Children & Adolescents, Paediat Gastronentrol Hepatol, Tubingen, Germany

[3] Childrens Hosp Westmead, Dept Gastroenterol, Sydney, NSW, Australia

[4] Clin Univ St Luc, Gastropediat, Brussels, Belgium

[5] Royal Childrens Hosp Melbourne, Dept Gastroenterol, Parkville, Vic, Australia

[6] Hop Univ Necker Enfants Malades, Gastroenterol Hepatol Nutr Unit, Paris, France

[7] Childrens Mem Hlth Inst, Dept Gastroenterol Hepatol Feeding Disorders & Pe, Warsaw, Poland

[8] Hosp Univ La Paz, Serv Hepatol & Transplante, Madrid, Spain

[9] Shire Int GmbH, Zug, Switzerland

[10] Shire Pharmaceut, Lexington, MA USA

[11] Shire Pharmaceut, Cambridge, MA USA

[12] Stanford Univ, Palo Alto, CA 94304 USA

[13] Reneo Pharmaceut, San Diego, CA USA

[14] Kings Coll London, Inst Liver Studies, London, England

来源：

关键词：

D O I：

10.1016/S0618-8278(19)30211-7

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

PS-193

引用

页码：E119 / E120

页数：2