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Casimersen (AMONDYS 45™): An Antisense Oligonucleotide for Duchenne Muscular Dystrophy
Casimersen (AMONDYS 45™) is an FDA-approved antisense oligonucleotide therapy for Duchenne muscular dystrophy (DMD) patients whose mutations allow exon 45 skipping. This treatment increases dystrophin production, stabilizing muscle function. Approved in 2021, Casimersen has shown promising results in the ESSENCE trial, significantly boosting dystrophin levels. While it is not a cure, this therapy offers hope for approximately 8% of DMD patients with exon 45 mutations. Clinical trials continue to assess long-term benefits and potential side effects, particularly concerning kidney health.
