Pediatric neurologist Dr. John Brandsema discusses the ongoing research trials for gene therapy relating to Duchenne Muscular Dystrophy (DMD) and the challenges associated with effective approaches to treatment. Learn more about what's on the horizon for the future of gene therapy in DMD.
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This education is brought to you by the France Foundation and supported by an educational grant from Sarepta Therapeutics Inc. All information is intended for healthcare professionals.
Future of DMD Gene Therapy
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DMDICNMDDuchenneMuscular DystrophyServaisNeuromuscularGeneticDystrophingene therapyphenotypesneuromuscular disordersDuchenne Muscular Dystrophyanti-inflammatorygene mutationsnovel therapieshealthcarediseaserare diseasechronic weaknesschronic illnessclinical trialsminidystrophinmicrodystrophingenetic disorderstransgeneanti-transgeneShiehnewborn screeningRUSPWMSBiomarkersimmune response