The aim of the International MLD Registry Project is to capture the natural history as well as the clinical course of MLD during supportive care and intervention in a manner that can be used to further advance therapies and eventually find a cure for MLD. The goal is to establish a quantitative description of clinical variability in patients who carry the confirmed diagnosis of MLD.
The Registry will strive for a comprehensive collection of data, including all subtypes of MLD. The data will encompass retrospective as well as prospective data. All data will be deidentified and entered into a centralized database. The Registry subscribes to defined standards of quality assurance and aims for optimal data entry, storage and analysis.
Metachromatic Leukodystrophy (MLD) is a rare disorder with a significant clinical heterogeneity. In order to capture the degree of variability a worldwide approach and international database is necessary. Further, a particular urgency arises as potential new therapies need to be evaluated and new clinical trials are contemplated.