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enrolling by invitation

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications

key information

study id #: NCT04240314

condition: Duchenne Muscular Dystrophy

status: enrolling by invitation


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