Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASO
Duchenne Muscular Dystrophy (DMD) is a devastating X-linked genetic disorder characterized by progressive muscle degeneration due to mutations in the dystrophin gene. This results in the absence or dysfunction of the dystrophin protein, leading to muscle weakness, loss of ambulation, respiratory iss...
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| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2024-01-01
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| Series: | Current Research in Toxicology |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2666027X24000355 |
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