Article

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy: A GRASP-LGMD study

2023-10-06

Abstract excerpt

<h4>Background: </h4> 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/design: The primary goal of this study is to evaluate the utility of a set of outcome measures on a wide range of...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 09f55285-f3cc-58d1-8dd1-4fe42c49d5c9
DOI: 10.21203/rs.3.rs-3370395/v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy: A GRASP-LGMD studyDOI 10.21203/rs.3.rs-3370395/v1Read

Defining clinical endpoints in limb girdle muscular dystrophy: a GRASP-LGMD study.Semantic proximity Read

Expert Panel Curation of 31 Genes in Relation to Limb Girdle Muscular DystrophySemantic proximity Read

Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies.Semantic proximity Read

Expert panel curation of 31 genes in relation to limb girdle muscular dystrophy.Semantic proximity Read

LGMDD1 natural history and phenotypic spectrum: Implications for clinical trials.Semantic proximity Read

Natural history of LGMD 2A for delineating outcome measures in clinical trialsSemantic proximity Read

Assessment of the quality of life in patients with LGMD. The case of transportinopathy.Semantic proximity Read

Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpointsSemantic proximity Read

Landscape and Heterogeneity of Treatment Outcome Measures in Facioscapulohumeral Muscular Dystrophy: A Systematic Literature ReviewSemantic proximity Read

Seven autosomal recessive limb-girdle muscular dystrophies in the Brazilian population: from LGMD2A to LGMD2G.Semantic proximity Read

Improving Measurement and Priorities for Assessment in Clinical Trials of childhood-onset Dystonia International Core Outcome Set (Dystonia IMPACT-COS): A protocolSemantic proximity Read

Correlations between clinical severity, genotype and muscle pathology in limb girdle muscular dystrophy type 2A.Semantic proximity Read