Sarepta announces completion of the confirmatory trial commitment for its ultra-rare disease PMO therapies AMONDYS 45 and VYONDYS 53: While the ESSENCE study did not achieve statistical significance ...
AMONDYS 45 (casimersen) uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 45 of dystrophin pre-mRNA, resulting in exclusion ...
– Following feedback from FDA, Company intends to submit supplemental new drug applications to FDA by the end of April 2026 requesting conversion to traditional approval Sarepta requested a meeting ...
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