Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II (MPS II): A phase 1/2 study

被引：0

作者：

Muenzer, Joseph ^{[1
]}

Burton, Barbara K. ^{[2
]}

Harmatz, Paul ^{[3
]}

Rajan, Deepa ^{[4
]}

Jones, Simon ^{[5
]}

van den Hout, Hannerieke J. M. ^{[6
]}

Mitchell, John J. ^{[7
]}

Troyer, Matt ^{[8
]}

Engmann, Natalie ^{[8
]}

Caprihan, Rupa ^{[8
]}

Bhalla, Akhil ^{[8
]}

Zubizarreta, Imanol ^{[9
]}

Weichert, Arlette ^{[8
]}

Chin, Peter ^{[8
]}

Ho, Carole ^{[8
]}

机构：

[1] Univ North Carolina Chapel Hill, Chapel Hill, NC USA

[2] Northwestern Univ, Feinberg Sch Med, Chicago, IL USA

[3] UCSF, Benioff Childrens Hosp, Oakland, CA USA

[4] UPMC, Childrens Hosp Pittsburgh, Pittsburgh, PA USA

[5] St Marys Hosp, Manchester, England

[6] Erasmus Univ, Med Ctr, Rotterdam, Netherlands

[7] McGill Univ, Hlth Ctr, Montreal, PQ, Canada

[8] Denali Therapeut, South San Francisco, CA USA

[9] Denali Therapeut, Zurich, Switzerland

来源：

关键词：

D O I：

10.1016/j.ymgme.2024.108864

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

237

引用

页码：85 / 86

页数：2