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
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author Heslop, Emma
Turner, Cathy
Irvin, Anna
Muntoni, Francesco
Straub, Volker
Guglieri, Michela
author_facet Heslop, Emma
Turner, Cathy
Irvin, Anna
Muntoni, Francesco
Straub, Volker
Guglieri, Michela
author_sort Heslop, Emma
collection PubMed
description • 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.
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spelling pubmed-75645102020-10-16 Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead Heslop, Emma Turner, Cathy Irvin, Anna Muntoni, Francesco Straub, Volker Guglieri, Michela Neuromuscul Disord Workshop Report • 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. Pergamon Press 2021-01 2020-10-16 /pmc/articles/PMC7564510/ /pubmed/33158687 http://dx.doi.org/10.1016/j.nmd.2020.10.001 Text en
spellingShingle Workshop Report
Heslop, Emma
Turner, Cathy
Irvin, Anna
Muntoni, Francesco
Straub, Volker
Guglieri, Michela
Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead
title Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead
title_full Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead
title_fullStr Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead
title_full_unstemmed Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead
title_short Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead
title_sort gene therapy in duchenne muscular dystrophy: identifying and preparing for the challenges ahead
topic Workshop Report
url 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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