Clinical trial preparedness in facioscapulohumeral dystrophy: Outcome measures and patient access
Author:
Publisher
Elsevier BV
Subject
Genetics(clinical),Clinical Neurology,Neurology,Pediatrics, Perinatology, and Child Health
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3. Facioscapulohumeral muscular dystrophy;Tawil;Muscle Nerve,2006
4. Facioscapulohumeral muscular dystrophy and DUX4: breaking the silence;der Maarel;Trends Mol Med,2011
5. Evolutionary conservation of a coding function for D4Z4, the tandem DNA repeat mutated in facioscapulohumeral muscular dystrophy;Clapp;Am J Hum Genet,2007
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1. A Targeted Approach for Evaluating DUX4-Regulated Proteins as Potential Serum Biomarkers for Facioscapulohumeral Muscular Dystrophy Using Immunoassay Proteomics;Journal of Neuromuscular Diseases;2023-11-07
2. Facioscapulohumeral muscular dystrophy: the road to targeted therapies;Nature Reviews Neurology;2023-01-10
3. Facioscapulohumeral Muscular Dystrophy;CONTINUUM: Lifelong Learning in Neurology;2022-12
4. A Targeted Approach for Evaluating DUX4-Regulated Proteins as Potential Serum Biomarkers for Facioscapulohumeral Muscular Dystrophy Using Immunoassay Proteomics;2022-07-26
5. Reliability and validity of the FSHD-composite outcome measure in childhood facioscapulohumeral dystrophy;Neuromuscular Disorders;2021-08
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