From Failure to Meet the Clinical Endpoint to US Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis

被引：18
作者：
van Roon-Mom, Willeke [1 ,2 ]
Ferguson, Chantal [3 ]
Aartsma-Rus, Annemieke [1 ,2 ]
机构：
[1] Leiden Univ, Dutch Ctr RNA Therapeut, Dept Human Genet, Med Ctr, Leiden, Netherlands
[2] Leiden Univ, Dept Human Genet, Med Ctr, Leiden, Netherlands
[3] UMass Chan Med Sch, RNA Therapeut Inst, Worcester, MA USA
来源：
NUCLEIC ACID THERAPEUTICS | 2023年 / 33卷 / 04期
关键词：
SPINAL MUSCULAR-ATROPHY; NUSINERSEN; SURVIVAL; TRIAL;
D O I：
10.1089/nat.2023.0027
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
[No abstract available]
引用
收藏
页码：234 / 237
页数：4
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