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Entrada filed for regulatory authorization in the u.k Exon 45 is a hotspot for mutations in the dmd gene that cause duchenne muscular dystrophy, thus skipping exon 45 is a potential therapeutic approach which could potentially correct ∼9% of duchenne muscular dystrophy cases. In this review, we discuss exon skipping therapy and clinical studies that have been developed for 20 years.
Among them, exon skipping and microdystrophin gene transfer are promising therapeutic approaches based on the restoration or replacement of the dystrophin functionality. Amondys 45 is used to treat patients with duchenne muscular dystrophy (dmd) who have a confirmed mutation of the dystrophin gene that can be treated by skipping exon 45. Discover the dmd deletions that are theoretically amenable to exon 45 skipping.
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