Validation of the GMFC-MLD

GMFC-MLD 的验证

• 批准号: 10842097
• 负责人: FLORIAN S EICHLER
• 金额: $ 6.76万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-30 至 2025-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10842097
• 关键词: Case Report Form; Clinical Trials; Clinical Trials Network; Collaborations; Data; Data Collection; Database Management Systems; Databases; Development; Documentation; Electronic Health Record; Funding; Grant; Learning; Metachromatic Leukodystrophy; Natural History; Outcome Measure; Parents; Participant; Patient Outcomes Assessments; Preparation; Procedures; Research Project Grants; Statistical Data Interpretation; Supervision; Targeted Research; Validation; Work; college; data curation; data management; design; leukodystrophy; member; parent project; programs; skills; statistics; translational medicine; translational neuroscience

Description of the Research Project for the Supplementee The proposed work is distinct from, but within the scope of the existing GLIA-CTN funded-projects. This subproject focuses on metachromatic leukodystrophy (MLD), which is included broadly for data collection in Project 1. There is not an MLD-specific project as part of the GLIA-CTN. During the proposed work, Ms. Mutua will learn the steps on how to design and implement a rigorous natural history study from data base creation to development of standard operating procedures to data collection to statistical analysis. In her first year as a coordinator in the GLIA-CTN, Ms. Mutua was responsible for document preparation and data base management for the MLD natural history project. This involved the creation of clinical trial-style documentation, including case report forms and binders, for our natural history participants. She has worked closely with members of our data management core regarding data curation. She has performed statistical analysis on the related pilot data for this project with the close supervision of our statistics team. As such, she is deeply knowledgeable about implementation of a rigorous natural history study. She has studied statistics formally in college as well. The proposed work is well within the skills she has developed in the past year. This project supported independence and will nurture her passion for translational medicine and neuroscience.

补充者研究项目的描述 拟议的工作与现有 GLIA-CTN 资助项目不同，但在其范围内。这 子项目重点关注异染性脑白质营养不良 (MLD)，该项目广泛用于数据收集 项目 1. GLIA-CTN 中没有专门针对 MLD 的项目。在拟议的工作期间，Mutua 女士 将学习如何设计和实施严格的自然历史研究的步骤，从数据库创建到 制定标准操作程序，从数据收集到统计分析。在她作为一名 Mutua女士担任GLIA-CTN协调员，负责文件准备和数据库 MLD 自然历史项目的管理。这涉及临床试验风格文档的创建， 包括为我们的自然历史参与者提供的病例报告表和活页夹。她曾与 我们关于数据管理的数据管理核心成员。她对此进行了统计分析 在我们统计团队的密切监督下，该项目的相关试点数据。正因如此，她深深地 了解如何实施严格的自然历史研究。她正式学习了统计学 大学也是如此。拟议的工作完全符合她在过去一年中培养的技能。这个项目 支持独立，并将培养她对转化医学和神经科学的热情。