Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy
Cohen J, DeSimone A, Lek M, Lek A. Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy. Trends In Molecular Medicine 2020, 27: 123-137. PMID: 33092966, PMCID: PMC8048701, DOI: 10.1016/j.molmed.2020.09.008.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAnimalsBiomarkersClinical Studies as TopicCombined Modality TherapyDisease ManagementDisease SusceptibilityDrug DevelopmentDrug Evaluation, PreclinicalGenetic Predisposition to DiseaseHomeodomain ProteinsHumansMolecular Targeted TherapyMuscular Dystrophy, FacioscapulohumeralOxidation-ReductionTreatment OutcomeConceptsFacioscapulohumeral muscular dystrophyMuscular dystrophyClinical outcome measuresClinical trialsOutcome measuresTherapeutic approachesMost trialsMolecular therapyEarly trialsTherapeutic developmentTrialsCommon typeCausal roleDystrophyTherapeutic discoveryFSHD researchCombined advancesTherapy