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Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a debilitating and fatal genetic disease affecting 1/5000 boys globally, characterized by progressive muscle breakdown and eventual death, with an average lifespan in the mid–late twenties. While no cure yet exists for DMD, gene and antisense therapies have been...

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Detalles Bibliográficos
Autores principales: Wilton-Clark, Harry, Yokota, Toshifumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10054484/
https://www.ncbi.nlm.nih.gov/pubmed/36986639
http://dx.doi.org/10.3390/pharmaceutics15030778