Editorial: Lessons Learned from Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation

被引：1

作者：

Mayhew, Anna G. ^{[1
,2
]}

Nelson, Leslie ^{[3
]}

Guglieri, Michela ^{[1
,2
]}

Willis, Tracey ^{[4
]}

机构：

[1] Newcastle Univ, Translat & Clin Res Inst, John Walton Muscular Dystrophy Res Ctr, Newcastle Upon Tyne, England

[2] Newcastle Hosp NHS Fdn Trust, Newcastle Upon Tyne, England

[3] Univ Texas Southwestern Med Ctr, Dept Phys Therapy, Dallas, TX USA

[4] NHS Fdn Trust, Robert Jones & Agnes Hunt Orthopaed Hosp, Oswestry, England

来源：

FRONTIERS IN GENETICS | 2022年 / 13卷

关键词：

neuromuscular disorders (NMD); clinical trial; clinical outcome assessments; duchenne muscular dystrophy (DMD); spinal muscular atrophy (SMA); remote assessment;

D O I：

10.3389/fgene.2022.840074

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

引用

页数：2

共 16 条

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