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Duchenne Muscular dystrophy

Duchenne muscular dystrophy

A genetic muscle disease caused by DMD variants that disrupt production of functional dystrophin.

Overview

EXPLORE BY LEVEL

Duchenne muscular dystrophy is a genetic condition in which muscle cells do not make enough working dystrophin, a protein that helps support muscle fibers.

Quick Facts

AT A GLANCE
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See how Duchenne Muscular dystrophy links to other biological records.

Open network
ProcessExon Skippingmay be addressed by
All 1 Structured Connections
Duchenne Muscular dystrophy · DiseaseMAY BE ADDRESSED BY →Exon Skipping · Process

Recommended Reading

Trusted references for learning more about Duchenne Muscular dystrophy.

reviewPharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an updatePubMed · PMID 34797383
Research Claims

DMD is genetically heterogeneous. Exon-skipping eligibility and outcome depend on the specific variant, reading-frame consequences, drug chemistry, tissue exposure, and clinical evidence.