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The Natural History Of Metachromatic Leukodystrophy Study

The Natural History OMetachromatic Leukodystrophy (HOME) Study, hosted by NORD’s IAMRARE™ Registry Program, represents an opportunity to address an area of unmet need, providing dynamic data collection and a novel framework for building regulatory-grade rare disease natural history studies incorporating patient-reported information. 

The HOME Study enables patients and caregivers to virtually contribute directly to research from the comfort and safety of their home, without the demands and challenges of traveling to a study site. The goals of the study are to enhance understanding of metachromatic leukodystrophy, inform methods for building natural history studies to serve as external controls, reduce the burden for patient participation in clinical trials, and provide innovative methods for the use of natural history study data to help accelerate therapeutic drug development and FDA decision-making.   

 

This study is sponsored by NORD with support from FDA CBER FDABAA-19-00123-A3/BAA PMWP#109