Article

Mathematical modeling of the progression of Duchenne Muscular Dystrophy in San Diego in an era of allele specific oligonucleotide therapy

2024-04-19

Abstract excerpt

<title>Abstract</title> <p>Duchenne muscular dystrophy (DMD) is a relatively common, severe, progressive, X-linked muscle disorder caused by pathogenic variation in the dystrophin gene. Affected status can be determined at birth, 3-5 years before symptom onset. As effective corticosteroid and genetic therapies are becoming available for DMD, predictive models of DMD progression are needed to guide individualized...

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Literature Corpus work: 795662c0-1777-5d80-bcf4-fd391921cc33
DOI: 10.21203/rs.3.rs-4049883/v1

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