Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASO

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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Bibliographic Details
Main Authors: Umme Sabrina Haque, Melissa Kohut, Toshifumi Yokota
Format: Article
Language:English
Published: Elsevier 2024-01-01
Series:Current Research in Toxicology
Subjects:
DMD
Eteplirsen
Golodirsen
Viltolarsen
Casimersen
SRP-5051
Online Access:http://www.sciencedirect.com/science/article/pii/S2666027X24000355
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