Article

Brogidirsen and Exon 44 Skipping for Duchenne Muscular Dystrophy: Advances and Challenges in RNA-Based Therapy

2025-05-26

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a severe inherited muscle-wasting disorder associated with severe morbidity and mortality globally. Current treatment options have improved the quality of life of patients, but these treatments are only palliative. There is a need for more DMD treatment options. Antisense oligonucleotide (ASO) therapies have emerged as a promising personalized treatment option for patient group...

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Literature Corpus work: 2403f903-6f7a-5e87-bdd3-cb4120429cdb
DOI: 10.20944/preprints202505.1948.v1

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