An evaluation of onasemnogene abeparvovec for spinal muscular atrophy (SMN1)

被引：0

作者：

Waldrop, Megan A. ^{[1
,2
,3
]}

Connolly, Anne M. ^{[1
,2
,3
]}

Mendell, Jerry R. ^{[1
,2
,3
]}

机构：

[1] Nationwide Childrens Hosp, Abigail Wexner Res Inst, Ctr Gene Therapy, Columbus, OH USA

[2] Ohio State Univ, Dept Pediat, Wexner Med Ctr, Columbus, OH 43210 USA

[3] Ohio State Univ, Dept Neurol, Wexner Med Ctr, Columbus, OH 43210 USA

来源：

EXPERT OPINION ON ORPHAN DRUGS | 2021年 / 9卷 / 7-10期

关键词：

Gene therapy; onasemnogene abeparvovec; spinal muscular atrophy; GENE-REPLACEMENT THERAPY; NATURAL-HISTORY; COPY NUMBER; SMA; DELIVERY; MODEL; MICE;

D O I：

10.1080/21678707.2021.2003778

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

Introduction Onasemnogene abeparvovec is the first systemic gene replacement therapy approved by the FDA for any inherited condition and is the second FDA-approved genetic therapy for 5q spinal muscular atrophy. Areas covered We discuss the design and preclinical development of onasemnogene abeparvovec, along with clinical trial and real-world data focusing on efficacy and safety. Expert Opinion Although onasemnogene abeparvovec is strikingly effective for the treatment of 5q SMA, it is only approved for use under the age of 2 years and there are also two other FDA-approved molecular-based treatments. Many questions remain in terms of treatment selection, possibility of dose optimization, and combinational therapies.

引用

页码：199 / 204

页数：6

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