MARALIXIBAT LEADS TO SIGNIFICANT IMPROVEMENT IN CHOLESTATIC PRURITUS FOR CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS DUE TO TJP2 OR MYO5B DEFICIENCY: DATA FROM THE MARCH-PFIC TRIAL

被引：0

作者：

Miethke, Alexander G. ^{[1
]}

Porta, Gilda ^{[2
]}

Moukarzel, Adib ^{[3
]}

Lin, Chuan-Hao ^{[4
]}

D'Agostino, Daniel ^{[5
]}

Mogul, Douglas B. ^{[6
]}

Nunes, Tiago ^{[6
]}

Aguilar, Raul ^{[6
]}

Vig, Pamela ^{[6
]}

Chiou, Fang Kuan ^{[7
]}

Thompson, Richard J. ^{[8
]}

机构：

[1] Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH USA

[2] Univ Sao Paulo, Sch Med, Sao Paulo, Brazil

[3] Hotel Dieu France St Joseph Univ Hosp, Beirut, Lebanon

[4] Univ Southern Calif, Childrens Hosp Los Angeles, Los Angeles, CA USA

[5] Hosp Italiano Buenos Aires, Buenos Aires, Argentina

[6] Mirum Pharmaceut Inc, Foster City, CA USA

[7] Kk Womens & Childrens Hosp, Singapore, Singapore

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

来源：

HEPATOLOGY | 2023年 / 78卷

关键词：

D O I：

暂无

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

4604-C

引用

页码：S2095 / S2096

页数：2

共 6 条

[1] MARALIXIBAT LEADS TO SIGNIFICANT IMPROVEMENTS IN CHOLESTATIC PRURITUS FOR CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS WITHOUT A GENETIC DIAGNOSIS: DATA FROM THE MARCH-PFIC TRIAL
Horslen, Simon P.
Moukarzel, Adib
Miethke, Alexander G.
Mittal, Naveen
Ekong, Udeme
Kasi, Nagraj
Mogul, Douglas B.
Nunes, Tiago
Aguilar, Raul
Vig, Pamela
Gilmour, Susan M.
Thompson, Richard J.
HEPATOLOGY, 2023, 78 : S2096 - S2097
[2] Maralixibat leads to significant reductions in pruritus and improvements in sleep for children with progressive familial intrahepatic cholestasis: data from MARCH-PFIC
Thompson, Richard
Mogul, Douglas
Nunes, Tiago
Garner, Will
Vig, Pamela
Miethke, Alexander
JOURNAL OF HEPATOLOGY, 2023, 78 : S370 - S370
[3] Maralixibat leads to significant reductions in bilirubin for patients with progressive familial intrahepatic cholestasis: data from MARCH-PFIC
D'Antiga, Lorenzo
Miethke, Alexander
Mogul, Douglas
Nunes, Tiago
Garner, Will
Vig, Pamela
Thompson, Richard
JOURNAL OF HEPATOLOGY, 2023, 78 : S61 - S61
[4] IMPROVEMENTS IN PRURITUS WITH MARALIXIBAT ARE ASSOCIATED WITH IMPROVED QUALITY OF LIFE FOR PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: DATA FROM THE MARCH-PFIC TRIAL
Aqul, Amal A.
Miethke, Alexander G.
Lin, Chuan-Hao
Porta, Gilda
Sokal, Etienne M.
Mogul, Douglas B.
Nunes, Tiago
Baek, Marshall
Vig, Pamela
Baumann, Ulrich
Thompson, Richard J.
HEPATOLOGY, 2023, 78 : S2092 - S2093
[5] Analysis of safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: data from MARCH-PFIC
Miethke, Alexander
Moukarzel, Adib
Porta, Gilda
Esquer, Joshue Covarrubias
Czubkowski, Piotr
Ordonez, Felipe
Candusso, Manila
Aqul, Amal A.
Squires, Robert H.
Sokal, Etienne
D'Agostino, Daniel
Baumann, Ulrich
D'Antiga, Lorenzo
Kasi, Nagraj
Laborde, Nolwenn
Arikan, Cigdem
Lin, Chuan-Hao
Gilmour, Susan
Mittal, Naveen
Chiou, Fang Kuan
Horslen, Simon P.
Huber, Wolf-Dietrich
David-Feliciano, Susan
Chien, Elaine
Mogul, Douglas
Garner, Will
Nunes, Tiago
Lascau, Anamaria
Vig, Pamela
Hupertz, Vera
Gonzalez-Peralta, Regino
Ekong, Udeme
Hartley, Jane
Laverdure, Noemie
Ovchinsky, Nadia
Thompson, Richard
JOURNAL OF HEPATOLOGY, 2023, 78 : S387 - S388
[6] LONG-TERM MAINTENANCE OF RESPONSE AND IMPROVED LIVER HEALTH WITH MARALIXIBAT IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC): 2-YEAR DATA FROM THE MARCH-ON STUDY
Miethke, Alexander G.
Moukarzel, Adib
Porta, Gilda
Esquer, Joshue Covarrubias
Czubkowski, Piotr
Ordonez, Felipe
Candusso, Manila
Aqul, Amal A.
Squires, Robert H.
Sokal, Etienne
D'Agostino, Daniel
Baumann, Ulrich
D'Antiga, Lorenzo
Kasi, Nagraj
Laborde, Nolwenn
Arikan, Cigdem
Lin, Chuan-Hao
Gilmour, Susan M.
Mittal, Naveen
Chiou, Fang Kuan
Horslen, Simon P.
Huber, Wolf-Dietrich
Nunes, Tiago
Lascau, Anamaria
Longpre, Lara
Mogul, Douglas B.
Baek, Marshall
Vig, Pamela
Hupertz, Vera F.
Gonzalez-Peralta, Regino P.
Ekong, Udeme
Hartley, Jane
Laverdure, Noemie
Ovchinsky, Nadia
Thompson, Richard J.
HEPATOLOGY, 2024, 79 (02) : E75 - E76

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