PMID- 37919208 OWN - NLM STAT- MEDLINE DCOM- 20231204 LR - 20231217 IS - 1873-2364 (Electronic) IS - 0960-8966 (Linking) VI - 33 IP - 11 DP - 2023 Nov TI - 261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands. PG - 884-896 LID - S0960-8966(23)00734-4 [pii] LID - 10.1016/j.nmd.2023.09.008 [doi] AB - 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 17(th)-19(th) 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. CI - Copyright (c) 2023. FAU - Servais, Laurent AU - Servais L AD - MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK; Division of Child Neurology, Centre de Reference des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liege and University of Liege, Avenue de l'Hopital 1 4000 Liege, Belgium. Electronic address: laurent.servais@paediatrics.ox.ac.uk. FAU - Horton, Rebecca AU - Horton R AD - MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK. FAU - Saade, Dimah AU - Saade D AD - Department of Neurology, University of Iowa Carver College of Medicine, Iowa City, IA, USA. FAU - Bonnemann, Carsten AU - Bonnemann C AD - National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA. FAU - Muntoni, Francesco AU - Muntoni F AD - UCL Great Ormond Street Institute of Child Health, The Dubowitz Neuromuscular Centre, London, UK; National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK. CN - 261st ENMC workshop study group LA - eng PT - Clinical Conference DEP - 20231001 PL - England TA - Neuromuscul Disord JT - Neuromuscular disorders : NMD JID - 9111470 SB - IM MH - Humans MH - Netherlands MH - Dependovirus/genetics MH - *Muscular Dystrophy, Duchenne/genetics MH - *Neuromuscular Diseases/therapy MH - Genetic Therapy/adverse effects OTO - NOTNLM OT - Duchenne OT - Gene therapy OT - Immunity OT - Safety OT - Serious adverse event OT - Spinal miuscular atrophy OT - X linked myotubular myopathy COIS- Declaration of Competing Interest FM has received honoraria for scientific advisory boards from Novartis Gene Therapies Inc., Biogen, Novartis, PTC, Roche, Pfizer, Dyne Therapeutics and Sarepta. LS has received consulting fees from Sarepta, Pfizer, Roche, Affina, Renegex Bio, Audentes, Evox Therapeutics and Novartis. He has received honoraria from Sarepta, Roche, Audentes and Novartis. He is the secretary of the World Muscle Society. DS, RHH and CGB have no conflicts of interest to declare. FIR - Adjali, Dr Oumeya IR - Adjali DO FIR - Beggs, Dr Alan IR - Beggs DA FIR - Bharucha, Dr Diana IR - Bharucha DD FIR - Bonnemann, Dr Carsten IR - Bonnemann DC FIR - Braun, Dr Serge IR - Braun DS FIR - Byrne, Dr Barry IR - Byrne DB FIR - Corti, Dr Manuela IR - Corti DM FIR - Buj-Bello, Dr Ana IR - Buj-Bello DA FIR - Chamberlain, Dr Jeff IR - Chamberlain DJ FIR - Ferreiro, Dr Ana IR - Ferreiro DA FIR - Flanigan, Dr Kevin IR - Flanigan DK FIR - Germanenko, Mrs Olga IR - Germanenko MO FIR - Goemans, Dr Nathalie IR - Goemans DN FIR - Grant, Dr Daniel IR - Grant DD FIR - Hopkins, Dr Sam IR - Hopkins DS FIR - Horton, Dr Rebecca IR - Horton DR FIR - Kollb-Sielecka, Dr Marta IR - Kollb-Sielecka DM FIR - Le Guiner, Dr Caroline IR - Le Guiner DC FIR - Levy, Dr Dan IR - Levy DD FIR - Lek, Dr Angela IR - Lek DA FIR - Miller, Dr Weston IR - Miller DW FIR - Morris, Dr Carl IR - Morris DC FIR - Dreghici, Dr Roxana IR - Dreghici DR FIR - Muntoni, Dr Francesco IR - Muntoni DF FIR - Saade, Dr Dimah IR - Saade DD FIR - Servais, Dr Laurent IR - Servais DL FIR - Singh, Dr Teji IR - Singh DT FIR - Vroom, Drs Elisabeth IR - Vroom DE FIR - Wagner, Dr Kathryn IR - Wagner DK FIR - Van Ieperen, Mr Frank IR - Van Ieperen MF EDAT- 2023/11/03 00:42 MHDA- 2023/12/04 12:43 CRDT- 2023/11/02 22:58 PHST- 2023/08/15 00:00 [received] PHST- 2023/09/17 00:00 [revised] PHST- 2023/09/24 00:00 [accepted] PHST- 2023/12/04 12:43 [medline] PHST- 2023/11/03 00:42 [pubmed] PHST- 2023/11/02 22:58 [entrez] AID - S0960-8966(23)00734-4 [pii] AID - 10.1016/j.nmd.2023.09.008 [doi] PST - ppublish SO - Neuromuscul Disord. 2023 Nov;33(11):884-896. doi: 10.1016/j.nmd.2023.09.008. Epub 2023 Oct 1.