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Sarepta's Amondys 45 (casimersen) Receives the US FDA's Approval for DMD in Patients Amenable to Skipping Exon 4

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Sarepta's Amondys 45 (casimersen) Receives the US FDA's Approval for DMD in Patients Amenable to Skipping Exon 4

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  • The approval is based on ESSENCE trial assessing Amondys 45 vs PBO in patients with DMD with a confirmed mutation amenable to exon 45 skipping. The study is expected to conclude in 2024
  • The study resulted in a significant increase in dystrophin production in skeletal muscle observed in patients treated with Amondys 45. Consistent with the accelerated approval- the continued approval of Amondys 45 may contingent on confirmation of clinical benefit in a confirmatory trial.
  • Amondys 45 is the third RNA exon-skipping treatment for DMD approved in the US. The company will initiate the commercial distribution of therapy in the US imminently

 ­ Ref: Sarepta | Image: Sarepta

Click here to­ read the full press release 

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