The National Organization for Rare Disorders (NORD) invites the Metachromatic Leukodystrophy (MLD) community to attend a public webinar to learn more about our HOME Study, Natural History Of Metachromatic Leukodystrophy. The HOME Study’s patient-centered design provides an opportunity for the MLD community to virtually contribute to research from the comfort and safety of home, without the demands and challenges of traveling to a study site. The goals of this FDA-funded project 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.
Questions from members of the rare community will be collected during the registration period as well as during the event and addressed during the webinar.
Topics will include:
Intro to the HOME Study: how the study was designed and what it aims to accomplish)
Why the HOME Study is so critical for the MLD community and the rare community as a whole
How to enroll in the HOME Study and what participation will entail
This webinar is intended for MLD patients, caregivers, and advocates, and is also open to the public.
For those unable to join live, this webinar will be recorded and a viewing link sent to all registrants for on-demand viewing.