Viltolarsen: From Preclinical Studies to FDA Approval
Rohini Roy Roshmi, Toshifumi Yokota
Topics & Concepts
MedicineClinical trialExon skippingDuchenne muscular dystrophyFood and drug administrationDystrophinClinical researchPopulationMuscular dystrophyExonBioinformaticsOncologyPharmacologyInternal medicineGeneGeneticsBiologyAlternative splicingEnvironmental healthMuscle Physiology and DisordersCardiomyopathy and Myosin StudiesCRISPR and Genetic Engineering