The Natural History Of Metachromatic Leukodystrophy Study
Contribute directly to research from the comfort of your home! NORD’s Natural History Of Metachromatic Leukodystrophy (HOME) Study is an initiative that expands patient and caregiver engagement in clinical research.
Requirements and benefits
Enrolling in this study takes less than 30 minutes and only requires quick updates on a quarterly or “as needed” basis. Anyone with a diagnosis of metachromatic leukodystrophy (MLD) may join, regardless of prior treatment of any kind, current trial involvement, or current involvement with other registries or natural history studies. Those with an MLD diagnosis, regardless of whether they developed symptoms or not, are eligible to participate. Newly diagnosed patients are encouraged to join, and their input is important to inform all disease stages. Once enrolled, your family will be mailed a brand-new tablet which you can use to engage with the study and for your own personal use.
Data collected from this study will help support clinical trials and may help accelerate regulatory approvals for therapeutic treatment of MLD. Help drive MLD research by enrolling today!
This study is developed by the National Organization for Rare Disorders (NORD), in partnership with the US Food & Drug Administration (FDA) and funded through award #75F40119C10091 to NORD from the FDA.