The South Carolina Muscular Dystrophy Surveillance, Tracking, and Research Network (SC MD STARnet) - Component A

抽象组分A 南卡罗来纳州肌营养不良监测、跟踪和研究网络 (SC MD STARnet）是州卫生部与 州的旗舰大学（南卡罗来纳州，南加州大学）。有两 该项目的总体目标：（1）维持一个以人口为基础的动态 肌营养不良症（MD）的监测系统，以及（2）进行持续分析 MD患者的健康状况、卫生保健利用和公共卫生影响。 我们将估计患病率、生存率，并跟踪护理的临床指标， 了解诊断的患病率、疾病进展、临床护理和健康状况。 此外，我们将设计，传播和分析调查的答复， 进行焦点小组/访谈，以了解MD患者的生活经历。 我们将对符合条件的MD病例进行纵向、基于人群的监测， 强直性肌营养不良（DM）、肩束肱型（FSHD）、肢带型（LGMD）， 先天性（CMD）、Emery-Dreirfuss（EDMD）和远端MD。我们将按照 Duchenne/Becker MD队列，以确定患病率是否发生变化， 生存和描述进步和关怀。我们还将开发研究方法， 工具，包括调查和采访，并与其他网站和 CDC/NCBDDD。所有这些工作都将在我们与MD建立伙伴关系的同时完成 合作伙伴/利益相关者以及我们与其他机构的合作。进行的目的 通过监测确定的亚群研究是为了解决 知识差距（例如，生活质量，怀孕和生育问题，以及自我护理问题， 等）。我们将分析和发表至少5篇手稿，并提出有关的发现。 监测和研究至少举行5次全国临床医生会议，保健 提供者、倡导者和其他相关方。

Abstract Component A South Carolina Muscular Dystrophy Surveillance, Tracking, and Research Network (SC MD STARnet) is a collaboration between the state health department and the state’s flagship university (University of South Carolina, USC). There are two overarching aims of this project: (1) is to maintain a dynamic population-based surveillance system for muscular dystrophy (MD), and (2) to conduct ongoing analyses of the health status, health care utilization, and public health impact of people with MD. We will estimate prevalence, survival, and track clinical indicators of care in order to understand diagnosed prevalence, disease progression, clinical care, and health status. In addition, we will design, disseminate, and analyze responses from survey(s) and conduct focus groups/interviews to understand the lived experience of people with MD. We will conduct longitudinal, population-based surveillance on eligible MD cases with Myotonic Dystrophy (DM), Fascioscapulohumeral (FSHD), Limb-girdle (LGMD), congenital (CMD), Emery-Dreirfuss (EDMD) and distal MD. We will follow the Duchenne/Becker MD cohort to determine if there are changes in prevalence and survival and to describe progress and care. We will also develop research methods and tools, including surveys and interviews, and conduct research with other sites and CDC/NCBDDD. All this work will be done while we build partnerships with MD partners/stakeholders and as we collaborate with other agencies. The aim of conducting research with the subpopulations identified through surveillance is to address knowledge gaps (eg. Quality of life, pregnancy and fertility issues, and self-care issues, etc.). We will analyze and publish at least 5 manuscripts and present findings about the surveillance and research studies at least 5 national meetings of clinicians, health care providers, advocates, and other interested parties.