261st ENMC workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofdorp, The Netherlands

Laurent Servais; Rebecca Horton; Dimah Saade; Carsten Bonnemann; Francesco Muntoni

doi:10.1016/j.nmd.2023.09.008

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261st ENMC workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofdorp, The Netherlands

Journal article

Peer reviewed

261st ENMC workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofdorp, The Netherlands

Laurent Servais, Rebecca Horton, Dimah Saade, Carsten Bonnemann and Francesco Muntoni

Neuromuscular disorders : NMD, Vol.33(11), pp.884-896

11/2023

DOI: 10.1016/j.nmd.2023.09.008

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Abstract

•I am not sure that this is needed for ENMC report- but will be happy to provide one Adeno-associated virus (AAV) gene therapies are demonstrating much promise in the area of Neuromuscular Disorders. There are now therapies in clinical trials or real-world use for several disorders including Spinal Muscular Atrophy and Duchenne Muscular Dystrophy. However, there have been several concerning reports of serious adverse events, including deaths. Reporting and monitoring of these is not consistent between trials. Therefore, a group of clinicians, investigators, industry and patient representatives met the weekend of 17th-19th June 2022 to discuss safety issues arising from the use of these therapies. The group shared information on safety events across a spectrum of AAV gene therapy products, both in clinical trials and commercial use. Patterns of serious adverse events were identified and the group discussed methods of identification and management of these as well as new ways of improving information sharing across industry in order to improve the safety of these promising treatments.

Gene Therapy

Safety

Duchenne

Immunity

Serious adverse event

Spinal Miuscular Atrophy

X linked Myotubular Myopathy

Details

Title: Subtitle: 261st ENMC workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofdorp, The Netherlands
Creators: Laurent Servais - University of Oxford
Rebecca Horton - University of Oxford
Dimah Saade - University of Iowa
Carsten Bonnemann - National Institute of Neurological Disorders and Stroke
Francesco Muntoni - Great Ormond Street Hospital
Resource Type: Journal article
Publication Details: Neuromuscular disorders : NMD, Vol.33(11), pp.884-896
DOI: 10.1016/j.nmd.2023.09.008
ISSN: 0960-8966
eISSN: 1873-2364
Publisher: Elsevier B.V
Language: English
Electronic publication date: 10/2023
Date published: 11/2023
Academic Unit: Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
Record Identifier: 9984473762802771

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