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

IF 3.2 4区医学 Q2 CLINICAL NEUROLOGY

Journal of neuromuscular diseases Pub Date : 2024-01-01 DOI:10.3233/JND-240002

Angela Lek, Evrim Atas, Brian Lin, Sharon E Hesterlee, Jordan K Abbott, Barry J Byrne, Carsten G Bönnemann

引用次数: 0

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.

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会议报告：2023 年肌肉萎缩症协会 "神经肌肉疾病基因疗法的安全性与挑战 "峰会。

本会议报告总结了 2023 年肌肉萎缩症协会（MDA）主办的基因疗法挑战峰会上的发言和讨论。涉及的主题包括神经肌肉疾病基因疗法临床转化的安全问题、缓解策略和实际考虑因素。列出的可行建议将有助于该领域的整体努力，实现神经肌肉疾病患者基因疗法的安全和有效转化。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of neuromuscular diseases Medicine-Neurology (clinical)

CiteScore

5.10

自引率

6.10%

发文量

102

期刊介绍： The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.