New Directions in Biology and Disease of Skeletal Muscle

骨骼肌生物学和疾病的新方向

• 批准号: 10400988
• 负责人: Elizabeth M McNally
• 金额: $ 1万
• 依托单位: NORTHWESTERN UNIVERSITY AT CHICAGO
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-09-01 至 2022-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10400988
• 关键词: Access to Information; Address; Advocacy; Biology; Biotechnology; Caring; Clinical Trials; Collaborations; Communities; Congresses; Data; Disabled Persons; Disease; Doctor of Philosophy; Drug Industry; Duchenne muscular dystrophy; Ensure; FDA approved; Family; Feedback; Funding; Gene therapy trial; Genes; Genetic Variation; Goals; Grant; Group Meetings; Human Genetics; Industry; Inflammatory Response; Inherited; Laboratories; Metabolism; Muscle; Muscular Dystrophies; Mutation; Myopathy; Neuromuscular Diseases; New York; Oral; Participant; Pathway interactions; Patient advocacy; Patients; Performance; Pharmaceutical Preparations; Pharmacologic Substance; Publications; Request for Proposals; Research; Research Personnel; Scientist; Series; Skeletal Muscle; Spinal Muscular Atrophy; Students; Testing; Time; TimeLine; Translational Research; Translations; Travel; Work; base; design; diversity and inclusion; doctoral student; exon skipping; gene therapy; improved; instructor; interest; meetings; micro-dystrophin; muscle strength; novel; novel therapeutics; posters; professor; programs; recruit; response; skeletal disorder; therapy development; unpublished works

PROJECT SUMMARY This is a new proposal requesting partial support for "The New Directions in Biology and Disease of Skeletal Muscle," to be held July 6-9, 2020. The meeting will convene at the New York Marriott Marquis in Times Square. The “New Directions” initiated in 2004, and this meeting has taken place every other year, making the 2020 meeting the 9th in the series. The goal of this meeting is to promote translational science for neuromuscular disease, especially the muscular dystrophies. In 2004, at the time of the first meeting, there were no FDA approved drugs for neuromuscular disease and a limited pipeline with limited interest from the pharmaceutical industry and biotech. With the purpose of this meeting to bring together stakeholders including academic laboratories, small and large pharmaceutical interests, and patient/family advocacy groups, this meeting has been instrumental in promoting the identification and translation of new targets for neuromuscular disease. This meeting was developed in response to the MD Care Act, which recognized a need for a neuromuscular disease focused meeting with specific emphasis on target identification and translation of basic discovery into clinical trials for those with neuromuscular disease. The New Directions meeting differs from other muscle biology-related meetings because of inclusion of industry and academic attendees as well as heavy emphasis on trainee participation. Exon skipping drugs were approved for Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA) in 2016 and 2017. Gene therapy for SMA in 2019 with approved as onasmemnogene abeparvovec, and there are multiple ongoing micro-dystrophin gene therapy trials for DMD. Based on this progress and on feedback from participants at the 2018 meeting, this next meeting will expand to other forms of inherited muscular diseases and focus on novel mechanisms for these disorders.

项目概要 这是一项新提案，要求部分支持“骨骼生物学和疾病的新方向” Muscle”将于2020年7月6日至9日举行。会议将在纽约时代广场万豪侯爵酒店举行 正方形。 “新方向”始于2004年，该会议每隔一年举行一次，使得 2020 年会议是该系列会议的第九次。本次会议的目标是促进转化科学 神经肌肉疾病，特别是肌营养不良症。 2004年，第一次会议时， FDA 还没有批准治疗神经肌肉疾病的药物，而且研发管线有限，人们的兴趣也有限 制药业和生物技术。本次会议的目的是将利益相关者聚集在一起，包括 学术实验室、小型和大型制药利益集团以及患者/家庭倡导团体，这 会议对于促进神经肌肉新靶标的识别和转化发挥了重要作用 疾病。这次会议是为了响应《医学博士护理法案》而召开，该法案认识到需要 以神经肌肉疾病为主题的会议，特别强调目标识别和基础知识的转化 将发现纳入针对神经肌肉疾病患者的临床试验中。新方向会议不同于 其他与肌肉生物学相关的会议，因为包括工业界和学术界的与会者以及 高度重视学员的参与。外显子跳跃药物被批准用于杜氏肌肉治疗 2016 年和 2017 年营养不良 (DMD) 和脊髓性肌萎缩症 (SMA)。2019 年 SMA 基因治疗 被批准为 onasmemnogene abeparvovec，并且有多种正在进行的微肌营养不良蛋白基因治疗 DMD 试验。基于这一进展以及 2018 年会议参与者的反馈，下一步 会议将扩展到其他形式的遗传性肌肉疾病，并重点关注这些疾病的新机制 失调。