An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy

Other illness

• Have known coagulation disorder

Lab values

• Are taking any prohibited medications
  • any approved exon skipping therapy within 12 weeks prior to baseline or with any gene therapy for the treatment of DMD at any time
  • anti-coagulants, anti-thrombotics, or anti-platelet agents
  • immunosuppressants

• Participants must have completed 351-201 without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study
• Currently receiving treatment with oral corticosteroids, on a stable dose regimen during 351-201, and must remain on a consistent dose regimen throughout 351-202 or 351-203 except for modifications to accommodate changes in weight
  • Transition to the equivalent dose of vamorolone is permitted in 351-202 where approved in participating countries.
• Willing and able to adhere to the study visit schedule and other protocol requirements
• Willing to use contraception (sexually mature males) throughout the study and for 90 days after the final dose, if sexually active
  • Contraceptive use by males should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• Willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure
• Willing and able to provide written, signed informed consent as parent or guardian after the nature of the study has been explained and prior to performance of any research-related procedure