Clinical Research Directory

Key Inclusion Criteria: * Ambulatory males with clinical and genetic diagnosis of DMD * Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping * 7 to 16 years of age at time of consent * TTR and NSAA assessment completed within the protocol specified parameters at Screening * On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable. Key Exclusion Criteria: * Previous treatment cell or gene therapy. * Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines). * Lab values outside of the protocol specified range at Screening * If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows: * Less than 1 month, for growth hormone and/or testosterone * Less than 6 months for givinostat