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New Directions in Biology and Disease of Skeletal Muscle

骨骼肌生物学和疾病的新方向

基本信息

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
项目类别：
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strength novel novel therapeutics posters professor programs recruit response skeletal disorder therapy development unpublished works

项目摘要

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.

项目总结