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

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.

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Bibliographic Details
Published in:Neuromuscular disorders : NMD Vol. 31; no. 1; pp. 69 - 78
Main Authors: Heslop, Emma, Turner, Cathy, Irvin, Anna, Muntoni, Francesco, Straub, Volker, Guglieri, Michela
Format: Journal Article Conference Proceeding
Language:English
Published: England Elsevier B.V 01-01-2021
Pergamon Press
Subjects:
Barriers
Collaboration
Duchenne muscular dystrophy
Gene therapy
Workshop Report
Gene therapy
Duchenne muscular dystrophy
Collaboration
Barriers
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Description
Summary:•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.
Bibliography:ObjectType-Conference Proceeding-1
SourceType-Conference Papers & Proceedings-1
content type line 25
ISSN:0960-8966
1873-2364
DOI:10.1016/j.nmd.2020.10.001