New York MDSTARnet Feasibility of Expansion to other Muscular Dystrophies

描述（由申请人提供）：肌营养不良症是一组异质性遗传性肌肉疾病，其特征是肌肉萎缩和无力，影响儿童和成人。在一项回顾性研究中，遗传性神经肌肉疾病的全球患病率估计为28.6/100，000和33.3/100，000，如果包括更罕见的疾病。作为一个群体，这些疾病涵盖了相当大比例的慢性疾病患者;然而，最近的研究，在美国的肌营养不良症的患病率是缺乏的。在两个资助周期中，肌营养不良监测、跟踪和研究网络（MD STARnet）已经确定并跟踪了杜氏和贝克尔肌营养不良症（DBMD）患者的健康状况。这个新的资助周期将寻求通过MD STARnet的扩展和修改来描述患有各种肌营养不良症的人群。该项目将评估使用MD STARnet现有基础设施扩展到其他类型肌营养不良症人群监测的可行性。 公共卫生相关性：利用肌营养不良监测、跟踪和研究网络（MD STARnet）的基础设施，该项目将寻求通过扩展和修改MD STARnet来表征患有各种肌营养不良症的人群。

DESCRIPTION (provided by the applicant): 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 cover 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 dystrophies (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. PUBLIC HEALTH RELEVANCE: Using the infrastructure of the Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet), this project will seek to characterize the population of individuals who have a variety of muscular dystrophies through the expansion and modification of MD STARnet.