Dystonia Coalition

肌张力障碍联盟

基本信息

批准号：
8330833
负责人：
HYDER A JINNAH
金额：
$ 128.44万
依托单位：
EMORY UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2009
资助国家：
美国
起止时间：
2009-09-30 至 2014-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8330833
关键词：
Address Affect Animals Area Basic Science Biocompatible Materials Biological Biological Markers Blepharospasm Body part Botulinum Toxins Cataloging Catalogs Cerebral cortex Cervical Dystonia Clinical Clinical Data Clinical Research Clinical Sciences Clinical Trials Collaborations Collection Commit Communication Communities Comorbidity Complement Complex Consensus Coping Skills Data Development Diagnosis Diagnostic Discrimination Disease Disease Progression Dystonia Elements Ensure Environment Epilepsy Europa Europe European Exhibits Face Focal Dystonias Foundations Functional disorder Funding Genes Glues Goals Grant Grouping Guanine Nucleotide Dissociation Inhibitors Hand Home environment Individual Industry Institution International Internet Larynx Lead Leg Letters Life Limb Dystonia Link Measures Medical Medical Research Methods Modeling Monitor Motor Movement Movement Disorders Multi-Institutional Clinical Trial Natural History Neurology New Agents North America Operative Surgical Procedures Ophthalmology Otolaryngology Outcome Parkinson Disease Pathogenesis Pathway interactions Patients Pattern Phenotype Physical Medicine Physiological Pilot Projects Play Population Posture Primary Dystonias Procedures Program Development Provider Publications Quality of life Rare Diseases Recruitment Activity Research Research Infrastructure Research Personnel Research Project Grants Resources Role Screening procedure Serum Severities Severity of illness Site Spastic Dysphonias Specialized Center Specimen Speech Therapy Sum Surrogate Markers Syndrome Testing Therapy Clinical Trials Time Torticollis Training Programs Translating Translations Treatment outcome United States National Institutes of Health Universities Validation Voice Disorders Washington Work alternative treatment arm base biobank career craniofacial design disability drug development drug discovery emotional reaction experience flexibility foot functional disability improved instrument interest meetings motor disorder multidisciplinary nervous system disorder neural circuit neuroimaging novel operation patient registry programs relating to nervous system repository response tool treatment response web site

项目摘要

DESCRIPTION (provided by applicant): The overall goal of the Dystonia Coalition is to develop a program to facilitate collaborations that will advance the pace of clinical research for the dystonias, a group of neurological disorders characterized by involuntary twisting movements and unnatural posturing. The Dystonia Coalition encompasses multiple investigators at different centers in North America and Europe, working together with several lay patient support organizations and research foundations on three Main Clinical Projects, a Pilot Projects Program, a Development (Training) Program, and an internet portal. Main Clinical Project 1 addresses the natural history of the most common forms of primary dystonia that affect specific body parts and collectively comprise approximately 80% of all dystonias: cervical dystonia (torticollis), laryngeal dystonia (spasmodic dysphonia), blepharospasm (peri-ocular regions), craniofacial dystonia (upper and lower face), and limb dystonias (hand/arm or foot/leg). Longitudinal clinical data will be linked with a biospecimen repository for DMA and serum to delineate potential biomarkers of disease status. Main Clinical Project 2 focuses on the development of comprehensive rating tools that can be used to monitor disease progression and responses to treatments for cervical dystonia, the most common of the focal dystonias. The methods used in Project 2 will serve as a guide for the development of similar tools for the other less common focal dystonias. Clinical Project 3 focuses on the development of practical clinical tools that can be used for definitive diagnosis of spasmodic dysphonia and discrimination from several related voice disorders. It too will serve as a guide for other focal dystonias where definitive diagnostic tools still are lacking, such as blepharospasm or limb dystonias. The Dystonia Coalition will provide a novel and valuable infrastructure and environment that will be used to accelerate clinical research in pursuit of improved treatment for all of the primary focal dystonias.

描述（由申请人提供）：肌张力障碍联盟的总体目标是制定一项计划，以促进合作，以提高肌张力障碍临床研究的步伐，肌张力纳斯是一组具有非自愿扭曲运动和不自然姿势的神经系统疾病。肌张力障碍联盟涵盖了北美和欧洲不同中心的多个研究人员，与几个外行患者支持组织和研究基金会合作，研究了三个主要的临床项目，一个试点项目计划，开发（培训）计划和互联网门户网站。主要的临床项目1介绍了影响特定身体部位的最常见形式的自然史，并共同占所有肌张力肌的约80％：颈肌张力障碍（corticollis）（torticollis），喉肌张力障碍（Spasmodicic）吞咽困难），骨痉挛（眼周区域），颅面肌张力障碍（上面和下面）和肢体肌张力障碍（手/手臂或脚/腿）。纵向临床数据将与DMA和血清的生物测量存储库相关联，以描绘疾病状态的潜在生物标志物。主要临床项目2着重于开发综合评级工具，可用于监测疾病进展和对宫颈肌甲抑制治疗的反应，颈肌甲抑制症是局灶性肌张力障碍中最常见的。项目2中使用的方法将作为开发其他不常见局灶性肌张力障碍工具的指南。临床项目3的重点是开发实用临床工具，这些工具可用于确切地诊断痉挛性吞咽困难，并歧视几种相关的语音疾病。它也将成为其他局灶性肌张力障碍的指南，其中仍然缺乏确定的诊断工具，例如骨痉挛或肢体肌张力障碍。肌张力障碍联盟将提供一种新颖而有价值的基础设施和环境，该基础设施和环境将用于加速临床研究，以改善所有主要局灶性抑制性肌张力障碍的治疗方法。