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

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

Abstract Background The Limb Girdle Muscular Dystrophies (LGMDs) are characterized by progressive weakness of the shoulder and hip girdle muscles as a result of over 30 different genetic mutations. This study is designed to develop clinical outcome assessments across the group of disorders. Methods/...

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Bibliographic Details
Main Authors: 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, On behalf of the GRASP-LGMD Consortium
Format: Article
Language:English
Published: BMC 2024-03-01
Series:BMC Neurology
Subjects:
Limb girdle muscular dystrophy
Muscular dystrophy
Clinical outcome assessments
Clinical trials
Therapeutic development
Online Access:https://doi.org/10.1186/s12883-024-03588-1

Internet

https://doi.org/10.1186/s12883-024-03588-1

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