Arizona Muscular Dystrophy Surveillance and Research Tracking Network (MD STARnet

! "#$%#&'()*$+,-.#/ 0.01！“0/”“'2）3#"，%.0”）45'-.6）7#"-.6）8#99（，：;）！！"#$$%&&'! "()%*+,-(.)!/ 0！ 目的1：开展以人群为基础的Duchenne/Becker型肌营养不良症的监测和研究 活动1.1.检查公共卫生实践中现有DBMD数据集的数据效用和质量。征得 其他MD STARnet站点通过新的或现有的委员会检查与数据相关的数据质量问题 为DBMD收集、管理和分析当前MD STARnet数据。 活动1.2.向CDC提供关于作为CDC一部分监测DBMD的研究中心经验的信息 MD STARnet的评估。 活动1.3.细化收集的DBMD患者临床数据变量的数量， 最多75-100个变量，产生可靠的相关信息，为公共卫生实践提供信息。 活动1.4.使用减少的数量对新的和随访的DBMD病例进行病历摘要 第一年的融资周期。 活动1.5.设计并进行访谈，以确定医疗过渡和其他影响生命阶段的问题 DBMD患者及其家人。 目标2：设计并实施基于人群的7种肌营养不良症监测 （强直性、先天性、肢带、Emery-Dreifuss、面肩肱、远端和眼咽） 活动2.1.通过评估现有海事处STARnet系统，修改或制定： 病例;（B）病例定义;（c）数据收集工具，以收集关于流行率、人口统计学、 （d）全面的质量控制程序，以解决数据质量问题 与数据收集、管理和分析有关的问题。 活动2.2.与当地诊所和利益相关者建立或保持伙伴关系 活动2.3.在Y2-Y3期间对另外7例肌营养不良症进行一次病历摘要 目标3：评估对9种肌营养不良症进行基于人群的监测的方法 活动3.1。在国家协调中心的领导下，与其他网站合作制作摘要 报告评估监测扩大到其他肌营养不良症。摘要应包括： 所用方法;（B）经验教训;（c）优势;（d）挑战;（e）成本;（f）障碍;（g）建议 进行肌肉萎缩症和罕见疾病监测计划。 目标4：利用MD STARnet收集的信息分析数据、发表文章和传播报告， 2002-2011 活动4.1。提出并领导至少三份手稿;在其他三份手稿上进行广泛合作。 活动4.2.每年至少参加一次在亚特兰大举行的PI会议，以便研究者介绍研究结果。

!"#$%#&'()*$+,-.#/'.0"1!"0/"'2)3#",%.0")45'-.6)7#"-.6)8#99(,:;)))!! "#$$%&&'!"()%*+,-(.)!/0! Objective 1: Conduct population-based surveillance and research of Duchenne/Becker muscular dystrophy Activity 1.1. Examine data utility and quality of existing DBMD data set for public health practice. Participate with other MD STARnet sites through a new or existing committee to examine data quality issues associated with data collection, management and analysis of current MD STARnet data for DBMD. Activity 1.2. Provide information to CDC regarding site experiences with surveillance for DBMD as part of a CDC assessment of MD STARnet. Activity 1.3. Refine the number of clinical data variables collected on individuals who have DBMD to include a maximum of 75-100 variables that yield reliable, relevant information that informs public health practice. Activity 1.4. Conduct medical record abstraction of new and follow-up DBMD cases using the reduced number of variables during year 1 of the funding period. Activity 1.5. Design and conduct an interview to determine healthcare transition and other life stage issues affecting individuals with DBMD and their families. Objective 2: Design and implement population-based surveillance on seven additional muscular dystrophies (Myotonic, Congenital, Limb-Girdle, Emery-Dreifuss, Facioscapulohumeral, Distal, and Oculopharyngeal) Activity 2.1. Through assessment of the existing MD STARnet system, modify or develop: (a) Protocols for ascertaining cases; (b) Case definitions; (c) Data collection instruments to collect information on prevalence, demographics, healthcare coverage, and locations of care; (d) Comprehensive quality control procedures to address data quality issues associated with data collection, management and analysis. Activity 2.2. Develop or maintain partnerships with local clinics and stakeholders Activity 2.3. Conduct medical record abstraction for seven additional muscular dystrophies once during Y2-Y3 Objective 3: Assess methodology for conducting population-based surveillance for the nine muscular dystrophies Activity 3.1. Under the leadership of the national coordinating center, collaborate with other sites to produce a summary report assessing the expansion of surveillance to other muscular dystrophies. The summary should include: (a) Methods used; (b) Lessons learned; (c) Strengths; (d) Challenges; (e) Costs; (f) Barriers; (g) Recommendations for conducting muscular dystrophy and rare disease surveillance programs. Objective 4: Analyze data, publish articles, and disseminate reports using information collected on MD STARnet from 2002-2011 Activity 4.1. Propose and lead a minimum of three manuscripts; extensively collaborate on three other manuscripts. Activity 4.2. Participate in at least one PI meeting per year in Atlanta, for investigators to present study findings.