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AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications
study id #: NCT04240314
condition: Duchenne Muscular Dystrophy
status: enrolling by invitation
purpose:Open-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.
intervention: scAAV9.U7.ACCA
mechanism of action: Exon-skipping to promote dystrophin production
results: https://clinicaltrials.gov/ct2/show/results/NCT04240314
last updated: February 01, 2020
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