Last updated on July 2020

Study of SRP-4045 and SRP-4053 in DMD Patients

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: DUCHENNE MUSCULAR DYSTROPHY | Muscular Dystrophy
  • Age: Between 7 - 13 Years
  • Gender: Male

Inclusion Criteria:

  • Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping
  • Stable dose of oral corticosteroids for at least 24 weeks
  • Intact right and left biceps or 2 alternative upper muscle groups
  • Mean 6MWT greater than or equal 300 meters and less than or equal to 450 meters
  • Stable pulmonary function: forced vital capacity (FVC) equal to or greater than 50% predicted

Exclusion Criteria:

  • Treatment with gene therapy at any time
  • Previous treatment with SMT C1100, PRO045 (BMN 045), PRO053 (BMN 053) or PRO051 (BMN 051) within 24 weeks prior to Week 1
  • Current or previous treatment with any other experimental treatment (other than deflazacort) within 12 weeks prior to Week 1
  • Major surgery within 3 months prior to Week 1
  • Presence of other clinically significant illness

Other inclusion/exclusion criteria may apply

Recruitment Status: Open

Brief Description Eligibility Contact Research Team

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