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2 trials match your criteria

A study to evaluate the long-term safety and effectiveness of BMN 351 in patients with Duchenne muscular dystrophy amenable to exon 51 skipping.

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Substances:BMN 351

Study of BMN 351 given by intravenous infusion to evaluate safety and tolerability in patients with Duchenne Muscular Dystrophy

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:BMN 351
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