The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European Experience

Antisense oligonucleotide (ASO) mediated exon skipping aims to reframe dystrophin transcripts for patients with Duchenne muscular dystrophy (DMD). Currently 4 ASOs have been approved by the Food and Drug Administration targeting exon 45, 51 and 53 based on low level dystrophin restoration. Additiona...

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Detalles Bibliográficos
Autores principales: Aartsma-Rus, Annemieke, De Waele, Liesbeth, Houwen-Opstal, Saskia, Kirschner, Janbernd, Krom, Yvonne D., Mercuri, Eugenio, Niks, Erik H., Straub, Volker, van Duyvenvoorde, Hermine A., Vroom, Elizabeth
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2023
Materias:
Commentary
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10200189/
https://www.ncbi.nlm.nih.gov/pubmed/36911945
http://dx.doi.org/10.3233/JND-221648

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10200189/
https://www.ncbi.nlm.nih.gov/pubmed/36911945
http://dx.doi.org/10.3233/JND-221648

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