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

NCT04240314 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

COMPLETED

Status

3

Enrollment

OTHER

Sponsor class

Conditions

Duchenne Muscular Dystrophy

Interventions

BIOLOGICAL: scAAV9.U7.ACCA

Sponsor

Megan Waldrop

Collaborators

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