罕见病联盟日本首届研讨会报告- 2023年7月18日,日本shonan健康创新园区。

IF 3.4 4区 医学 Q2 CLINICAL NEUROLOGY
Toshifumi Yokota, Naoto Inukai, Hiroyuki Shibasaki, Harumasa Nakamura, Shinnichi Yokota, Yoshitaka Nakamura, Rikako Yamauchi, Keiko Ishigami, Akitsu Hotta, Hideo Miki, Yoshitsugu Aoki
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引用次数: 0

摘要

日本罕见病联盟(RDCJ)是一个新近正式启动的跨部门倡议,旨在解决日本罕见病患者迫切和日益增长的需求,旨在支持广泛的罕见疾病,包括但不限于神经肌肉疾病。RDCJ于2023年7月18日在湘南健康创新园区举办了首届研讨会。本次研讨会旨在通过涉及工业界、政府、学术界、患者和社区的合作方法,解决罕见病的独特挑战。此次活动将这些利益攸关方聚集在一起,讨论日本罕见病研究和治疗的现状和未来方向。此次活动的特色是一系列演讲者和小组讨论,内容涉及药物开发状况、患者旅程和以患者为中心的医疗保健服务的未来。重点包括反义寡核苷酸治疗的主题演讲和以患者为中心的医疗保健、以人为本的医疗保健设计以及罕见疾病治疗的创新方法。本报告提供了研讨会的详细概述,每次会议的关键要点,以及RDCJ的未来方向。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Report on the rare disease consortium Japan inaugural symposium - July 18, 2023, shonan health innovation park, Japan.

The Rare Disease Consortium Japan (RDCJ) is a newly formalized cross-sector initiative launched to address the urgent and growing needs of individuals living with rare diseases in Japan, aiming to support a wide range of rare conditions, including-but not limited to-neuromuscular diseases. RDCJ hosted its inaugural symposium on July 18, 2023, at the Shonan Health Innovation Park. This symposium aimed to address the unique challenges of rare diseases through a collaborative approach involving industry, government, academia, patients, and the community. The event brought together these stakeholders to discuss the current state and future directions of rare disease research and treatment in Japan. The event featured a range of speakers and panel discussions on the state of drug development, patient journeys, and the future of patient-centered healthcare services. Highlights included a keynote on antisense oligonucleotide therapeutics and sessions on patient-centered healthcare, human-centered design in healthcare, and innovative approaches to rare disease treatment. This report provides a detailed overview of the symposium, the key takeaways from each session, and the future directions for RDCJ.

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来源期刊
Journal of neuromuscular diseases
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.
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