Shots:

• 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

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