Defining clinical endpoints in limb girdle muscular dystrophy: a GRASP-LGMD study

被引：0

作者：

Amy Doody

Lindsay Alfano

Jordi Diaz-Manera

Linda Lowes

Tahseen Mozaffar

Katherine D. Mathews

Conrad C. Weihl

Matthew Wicklund

Man Hung

Jeffrey Statland

Nicholas E. Johnson

机构：

[1] Virginia Commonwealth University,

[2] Nationwide Children’s Hospital,undefined

[3] Newcastle University,undefined

[4] University of California,undefined

[5] University of Iowa Carver College of Medicine,undefined

[6] Washington University,undefined

[7] UT Health San Antonio,undefined

[8] Roseman University,undefined

[9] University of Kansas Medical Center,undefined

来源：

BMC Neurology | / 24卷

关键词：

Limb girdle muscular dystrophy; Muscular dystrophy; Clinical outcome assessments; Clinical trials; Therapeutic development;

D O I：

暂无

中图分类号：

学科分类号：

摘要：

引用

共 239 条

[41] Yokota T(2013)Association of elbow flexor MRI fat fraction with loss of hand-to-mouth movement in patients with duchenne muscular dystrophy Muscle Nerve 48 343-undefined
[42] Zaidman CM(1969)The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study Arch Phys Med Rehabil 50 311-undefined
[43] Proud CM(2016)An objective and standardized test of hand function PLOS One 11 e0153095-undefined
[44] McDonald CM(undefined)Natural history of cardiac and respiratory involvement, prognosis and predictive factors for long-term survival in adult patients with limb girdle muscular dystrophies type 2 C and 2D Jefferies JL, editor undefined undefined undefined-undefined
[45] Lehman KJ(undefined)undefined undefined undefined undefined-undefined
[46] Goedeker NL(undefined)undefined undefined undefined undefined-undefined
[47] Mason S(undefined)undefined undefined undefined undefined-undefined
[48] Mendell JR(undefined)undefined undefined undefined undefined-undefined
[49] Rodino-Klapac LR(undefined)undefined undefined undefined undefined-undefined
[50] Rosales-Quintero X(undefined)undefined undefined undefined undefined-undefined