A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment

Participation Requirements

• Sex:

  MALE

• Eligible Ages:

  6 to 16

Participation Criteria

Inclusion Criteria:

* Confirmed diagnosis of DMD able to be corrected by skipping Exon 51
* Body weight at least 18kg at Screening
* Performance of Upper Limb (PUL) 2.0 entry score of at least 4 at Screening (assessing upper limb function in ambulant and non-ambulant individuals with DMD)

Exclusion Criteria:

* Known history or presence of any clinically significant conditions that may interfere with study safety assessments
* Treatment with any gene replacement therapy for the treatment of DMD at any time
* Current or recent systemic infection within 2 weeks prior to Screening or infection requiring IV antibiotics within 4 weeks prior to Screening
* Recent surgery requiring anesthesia within 3 months prior to Screening or expected surgery requiring general anesthesia during the study