BMN 351 Exon 51 Skipping Therapy for Duchenne Muscular Dystrophy: Phase 2 Extension 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

• Have known coagulation disorder
• 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

Not yet provided