Validation of the GMFC-MLD

补充对象研究项目说明 拟议的工作不同于现有的全球信托基金资助的项目，但在这些项目的范围之内。这 子项目的重点是异色性脑白质营养不良(MLD)，它被广泛包括在#年的数据收集中 项目1.作为GILA-CTN的一部分，没有专门针对MLD的项目。在拟议的工作期间，Mutua女士 我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。在她成为一名 Mutua女士是GILA-CTN的协调员，负责文件准备和数据库 MLD自然历史项目的管理。这包括创建临床试验风格的文档， 包括案例报告表和活页夹，供我们的自然历史参与者使用。她曾与 我们的数据管理核心成员与数据管理有关。她进行了统计分析，对 在我们统计团队的密切监督下，这一项目的相关试点数据。因此，她深深地 对实施严格的自然历史研究有一定的了解。年，她正式学习了统计学。 大学也是。拟议的工作完全符合她在过去一年中掌握的技能。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。

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.