Interim results of Transpher A, a multicentre, single-dose, phase 1/2 clinical trial of ABO-102 investigational gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)

被引：0

作者：

Flanigan, Kevin M. ^{[1
]}

Smith, Nicholas J. N. ^{[2
]}

Couce, Maria L. ^{[3
]}

Escolar, Maria ^{[4
]}

Truxal, Kristen V. ^{[1
]}

McBride, Kim L. ^{[1
]}

de Castro, Maria J. ^{[3
]}

Fuller, Maria ^{[5
]}

Paneda, A. ^{[6
]}

Ruiz, J. ^{[6
]}

del Campo, A. B. ^{[6
]}

Grachev, I. ^{[7
]}

机构：

[1] Nationwide Childrens Hosp, Columbus, OH USA

[2] Womens & Childrens Hosp, Adelaide, SA, Australia

[3] Hosp Clin Univ Santiago de Compostela, Santiago De Compostela, Spain

[4] Univ Pittsburgh, Med Ctr, Pittsburgh, PA USA

[5] Univ Adelaide, Adelaide, SA, Australia

[6] Abeona Therapeut, Madrid, Spain

[7] Abeona Therapeut, New York, NY USA

来源：

关键词：

D O I：

10.1016/j.ymgme.2021.11.099

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：S43 / S43

页数：1