GeneGene: DMD
Dystrophin gene (DMD) — exon 51 skipping
The DMD gene encodes dystrophin, the protein missing in Duchenne muscular dystrophy. Exon-skipping antisense oligonucleotides mask exon 51 during splicing so the cell produces a shortened but partially functional dystrophin, targeting the subset of patients whose mutations are amenable to exon 51 skipping.
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Drugs
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Trials tracked
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Resolved
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Indications
Drugs hitting this target
Trials targeting DMD / exon 51
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About DMD / exon 51
What is DMD / exon 51?
The DMD gene encodes dystrophin, the protein missing in Duchenne muscular dystrophy. Exon-skipping antisense oligonucleotides mask exon 51 during splicing so the cell produces a shortened but partially functional dystrophin, targeting the subset of patients whose mutations are amenable to exon 51 skipping.
Which drugs target DMD / exon 51?
Therapies acting on DMD / exon 51 tracked here include Zeleciment rostudirsen.