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Sarepta Announces Clinical Results from MOMENTUM study

Today, Sarepta shared an update for its investigational PPMO SRP-5051 clinical trial study. MOMENTUM, the name of their phase 2 clinical trial peptide phosphorodiamidate morpholino oligomer study is for patients with Duchenne Muscular Dystrophy amenable to skipping exon 51.

 

  • Results from the multiple-ascending dose trial demonstrate proof-of-concept for SRP-5051 and support continued dose escalation
  • At a total dose exposure approximately 10x lower than eteplirsen, SRP-5051 at 20 mgs/kg showed enhanced tissue exposure, greater exon skipping, and greater dystrophin production with no negative renal or other laboratory findings
  • These are the first clinical results from the Company’s peptide phosphorodiamidate morpholino oligomer (PPMO) technology, its next-generation chemistry platform
World Duchenne
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