Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

被引：29

作者：

George, Lindsey A. ^{[1
,2
,3
]}

Sullivan, Spencer K. ^{[4
]}

Rasko, John E. J. ^{[5
]}

Giermasz, Adam ^{[6
]}

Samelson-Jones, Benjamin J. ^{[7
]}

Ducore, Jonathan M. ^{[8
]}

Teitel, Jerome M. ^{[9
]}

McGuinn, Catherine E. ^{[10
]}

Runowski, Alexa R. ^{[7
]}

Wright, Fraser ^{[11
]}

Anguela, Xavier M. ^{[12
]}

High, Katherine A. ^{[12
]}

Rybin, Denis ^{[13
]}

Murphy, John E. ^{[14
]}

Rupon, Jeremy ^{[15
]}

机构：

[1] Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

[2] Childrens Hosp Philadelphia, Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA

[3] Univ Penn, Dept Pediat, Perelman Sch Med, Philadelphia, PA 19104 USA

[4] Mississippi Ctr Adv Med, Madison, MS USA

[5] Centenary Inst, Gene & Stem Cell Therapy Program, Camperdown, NSW, Australia

[6] Univ Calif Davis, Sacramento, CA 95817 USA

[7] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

[8] Univ Calif Davis, Hemophilia Treatment Ctr, Sacramento, CA USA

[9] St Michaels Hosp, Div Hematol & Oncol, Dept Med, Toronto, ON, Canada

[10] Weill Cornell Med Coll, New York, NY USA

[11] Stanford Univ, Sch Med, Ctr Definit & Curat Med, Stanford, CA 94305 USA

[12] Spark Therapeut, Philadelphia, PA USA

[13] Pfizer Inc, Cambridge, MA USA

[14] Pfizer Corp, Rare Dis Res Unit, Cambridge, MA USA

[15] Pfizer, Collegeville, PA USA

来源：

关键词：

D O I：

10.1182/blood-2019-124091

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

3347

引用

页数：4