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Article

The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European Experience

7 Mar 2023

Abstract excerpt

Antisense oligonucleotide (ASO) mediated exon skipping aims to reframe dystrophin transcripts for patients with Duchenne muscular dystrophy (DMD). Currently 4 ASOs have been approved by the Food and Drug Administration targeting exon 45, 51 and 53 based on low level dystrophin restoration. Additional studies to confirm functional effects are ongoing. Furthermore, efforts are ongoing to increase muscle specific...

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PMID: 36911945
DOI: 10.3233/jnd-221648

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