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.

被补充人研究项目说明 拟议的工作与现有的GLIA-CTN资助项目不同，但在其范围内。这 分项目的重点是异染性脑白质营养不良（MLD），这是广泛包括在数据收集， 项目1。作为GLIA-CTN的一部分，没有MLD特定项目。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，从数据库的创建， 制定从数据收集到统计分析的标准作业程序。在她作为一个 Mutua女士是GLIA-CTN的协调员，负责文件编制和数据库 管理MLD自然历史项目。这包括创建临床试验风格的文件， 包括病例报告表和活页夹，供我们的自然史参与者使用。她曾与 我们的数据管理核心成员关于数据策展。她对这些数据进行了统计分析， 在我们统计团队的密切监督下，为该项目提供相关试点数据。因此，她深深地 了解如何实施严格的自然历史研究。她在年正式学习统计学。 大学也是。这项拟议中的工作完全符合她在过去一年中所掌握的技能。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。