New York MDSTARnet Feasibility of Expansion to other Muscular Dystrophies

工程摘要 肌营养不良症是一组异质性遗传性肌肉疾病，其特征在于： 肌肉萎缩和虚弱，影响儿童和成人。的全球患病率 一项回顾性研究估计，遗传性神经肌肉疾病的发病率为28.6/100，000，如果更罕见，则为33.3/100，000 包括疾病。作为一个群体，这些疾病，涵盖了相当大比例的慢性 然而，最近在美国缺乏关于肌营养不良症患病率的研究。为 两个资助周期，肌肉营养不良监测，跟踪和研究网络（MD STARnet） 识别并跟踪杜氏和贝克肌营养不良症（DBMD）患者的健康状况。这 新的资助周期将寻求描述那些拥有各种肌肉发达的人的特征。 通过MD STARnet的扩展和修改来治疗营养不良。该项目将评估以下方面的可行性： 利用现有的MD STARnet基础设施，扩展到以人口为基础的其他类型的监测， 肌肉萎缩症

Project Abstract The muscular dystrophies are the heterogeneous group of inherited muscular disorders characterized by muscle wasting and weakness, and are affecting both children and adults. The worldwide prevalence of inherited neuromuscular diseases estimated in a review study was of 28.6/100,000 and 33.3/100,000 if rarer disorders are included. As a group, these disorders, covers a considerable proportion of patients with chronic disease, however, recent studies of prevalence of the muscular dystrophies in the United States are lacking. For two funding cycles, the Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet) has identified and tracked the health of patients with Duchennne and Becker muscular dystrophy (DBMD). This new funding cycle will seek to characterize the population of individuals who have a variety of muscular dystrophies through the expansion and modification of MD STARnet. The project will assess the feasibility of using the existing infrastructure of MD STARnet to expand to population-based surveillance for other types of muscular dystrophy.