Casimersen: First Approval
Matt Shirley
Topics & Concepts
Exon skippingDuchenne muscular dystrophyMedicineDystrophinExonAntisense therapyMorpholinoClinical trialOrphan drugGenetic enhancementBioinformaticsInternal medicineGeneGeneticsOligonucleotideBiologyAlternative splicingLocked nucleic acidZebrafishMuscle Physiology and DisordersProsthetics and Rehabilitation RoboticsCardiomyopathy and Myosin Studies