AOC 1044 Exon 44 Skipping Therapy for Duchenne Muscular Dystrophy: SAFARI44 Clinical Trial

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.

• Ambulatory males with clinical and genetic diagnosis of DMD
• 7 to 16 years of age at time of consent
• TTR and NSAA assessment completed within the protocol specified parameters at Screening

• 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