Meeting Report: 2023 Muscular Dystrophy Association Summit on ‘Safety and Challenges in Gene Therapy of Neuromuscular Diseases’-Reference-Cited by-同舟云学术

Meeting Report: 2023 Muscular Dystrophy Association Summit on ‘Safety and Challenges in Gene Therapy of Neuromuscular Diseases’

Published:2024-09-03 Issue:5 Volume:11 Page:1139-1160
ISSN:2214-3599
Container-title:Journal of Neuromuscular Diseases
language:
Short-container-title:JND

Author:

Lek Angela¹,Atas Evrim¹,Lin Brian¹,Hesterlee Sharon E.¹,Abbott Jordan K.²,Byrne Barry J.³,Bönnemann Carsten G.⁴

Affiliation:

1. Muscular Dystrophy Association, Chicago, IL, USA

2. Section of Allergy and Immunology, Children’s Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, USA

3. Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA

4. Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA

Abstract

This meeting report summarizes the presentations and discussions held at the summit on Challenges in Gene Therapy hosted by the Muscular Dystrophy Association (MDA) in 2023. Topics covered include safety issues, mitigation strategies and practical considerations pertaining to the clinical translation of gene therapies for neuromuscular disease. The listing of actionable recommendations will assist in overall efforts in the field to achieve safe and efficacious translation of gene therapies for neuromuscular disease patients.

Publisher

IOS Press

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