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Myelin Disorders Biorepository Project (MDBP) at the Biospecimen Exchange for Neurological Disorders (BioSEND)

神经疾病生物样本交换中心 (BioSEND) 的髓磷脂疾病生物储存库项目 (MDBP)

基本信息

批准号：
10850332
负责人：
FLORIAN S EICHLER
金额：
$ 14.34万
依托单位：
CHILDREN'S HOSP OF PHILADELPHIA
依托单位国家：
美国
项目类别：
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/10850332
关键词：
Academic Medical Centers Adherence Adrenoleukodystrophy Affect Age Alexander Disease Axon Biological Markers Cerebrospinal Fluid Child Clinical Data Clinical Research Clinical Trials Clinical Trials Network Collaborations Collection Communities Data Data Analytics Data Storage and Retrieval Development Disease Electronic Health Record Enrollment Ensure Funding Future Genomic DNA Genomics Genotype Goals Grant Health Hereditary Disease Indiana Individual Infrastructure Link Live Birth Maintenance Metachromatic Leukodystrophy Morbidity - disease rate Myelin National Center for Advancing Translational Sciences National Institute of Neurological Disorders and Stroke Natural History Neuroglia Outcome Outcome Measure Parents Patient Outcomes Assessments Patient advocacy Pediatric Hospitals Pelizaeus-Merzbacher Disease Phenotype Philadelphia Plasma Policies Population Heterogeneity Procedures Production RNA Rare Diseases Recurrence Research Resource Sharing Resources Sampling Serum Severity of illness Shipping Source Symptoms Texas Therapeutic Time Universities Urine Utah Work adjudication biobank biomarker development biomarker validation clinical research site clinical trial readiness data dissemination data management diagnostic biomarker functional outcomes genomic data industry partner interest leukodystrophy member metabolomics molecular targeted therapies monocyte mortality nervous system disorder next generation online resource parent project peripheral blood programs prospective pseudotoxoplasmosis syndrome recruit sample collection screening sex sustainable resource tool transcriptome sequencing

项目摘要

Summary of the Funded Grant or Project The parent RDCRN program is the Global Leukodystrophy Initiative Clinical Trial Network (GLIA-CTN). This program is in its first cycle (entering Y5) and is a collaboration of 8 US leukodystrophy centers. The primary aims focus on harmonization and curation of leukodystrophy natural history data using standard functional and patient reported outcome measures as well as electronic health records. The parent program supports the collection of clinical data related but does not include the proposed biobanking approaches. This application is expected to create the necessary infrastructure to include BioSEND prospectively in future GLIA-CTN activities.

已获资助的拨款或项目摘要 RDCRN的母项目是全球脑白质营养不良倡议临床试验网络（GLIA-CTN）。这该计划是在其第一个周期（进入Y 5），是一个合作的8个美国脑白质营养不良中心。主要目标专注于使用标准功能和患者模型协调和管理脑白质营养不良自然史数据报告的结果指标以及电子健康记录。父程序支持集合临床数据相关，但不包括拟定的生物库方法。该应用程序预计将创建必要的基础设施，以便在未来的GLIA-CTN活动中前瞻性地纳入BioSEND。