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Functional outcomes measured by North Star Ambulatory Assessment (NSAA) and serum creatine kinase were exploratory outcomes. Secondary outcomes included micro-dystrophin expression by Western blot and immunohistochemistry. Main Outcomes and Measures Safety was the primary outcome. Daily prednisolone, 1 mg/kg, started 1 day before gene delivery (30-day taper after infusion). Interventions A single dose of 2.0 × 10 14 vg/kg -dystrophin was infused through a peripheral limb vein. The first 4 patients who met eligibility criteria were enrolled, consisting of ambulatory male children with DMD without preexisting AAVrh74 antibodies and a stable corticosteroid dose (≥12 weeks). It began on November 2, 2017, with a planned duration of follow-up of 3 years, ending in March 2021.

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Objective To identify the 1-year safety and tolerability of intravenous -dystrophin in patients with DMD.ĭesign, Setting, and Participants This open-label, phase 1/2a nonrandomized controlled trial was conducted at the Nationwide Children’s Hospital in Columbus, Ohio. Importance Micro-dystrophin gene transfer shows promise for treating patients with Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype rh74 (rAAVrh74) and codon-optimized human micro-dystrophin driven by a skeletal and cardiac muscle-specific promoter with enhanced cardiac expression (MHCK7). Shared Decision Making and Communication.Scientific Discovery and the Future of Medicine.Health Care Economics, Insurance, Payment.

Clinical Implications of Basic Neuroscience.

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