Clinical potential of microdystrophin as a surrogate endpoint
J Boehler, Kristy J. Brown, Margaret Beatka, J. Patrick Gonzalez, Roxana Donisa Dreghici, Meghan Soustek-Kramer, Sharon McGonigle, Annie Ganot, T. N. Palmer, Caitlin Lowie, Jeffrey S. Chamberlain, Michael W. Lawlor, Carl Morris
Topics & Concepts
Surrogate endpointContext (archaeology)Clinical endpointClinical trialMedicineDuchenne muscular dystrophyMuscular dystrophyBiomarkerDystrophinExon skippingBioinformaticsIntensive care medicineInternal medicineBiologyExonGeneticsPaleontologyGeneAlternative splicingMuscle Physiology and DisordersVirus-based gene therapy researchTelomeres, Telomerase, and Senescence