Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead

• Despite the burden of gene therapy trials for DMD patients there is great enthusiasm. • Collaborating with relevant bodies (pharmacy) at an early stage can accelerate progress. • A hub and spoke model may be an option for delivering clinical trials and follow up care.

Detalles Bibliográficos
Autores principales: Heslop, Emma, Turner, Cathy, Irvin, Anna, Muntoni, Francesco, Straub, Volker, Guglieri, Michela
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Pergamon Press 2021
Materias:
Workshop Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7564510/
https://www.ncbi.nlm.nih.gov/pubmed/33158687
http://dx.doi.org/10.1016/j.nmd.2020.10.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7564510/
https://www.ncbi.nlm.nih.gov/pubmed/33158687
http://dx.doi.org/10.1016/j.nmd.2020.10.001

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