The National Organization for Rare Disorders is proud to announce the NORD Natural History of Metachromatic Leukodystrophy (HOME) Study, a brand-new initiative with support from FDA CBER! The HOME Study, hosted by NORD’s IAMRARE™ Program, 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 study will provide dynamic data collection and a new framework for building regulatory-grade rare disease natural history studies. Data collected securely through the system will provide researchers with the information they need to develop new diagnostic and treatment options. Participation is especially vital given the rarity of #leukodystrophy. Every patient experience is unique and an invaluable part of a natural history study.
“The HOME Study highlights the value of patient participation in research and therapy development, empowering MLD patients and caregivers to make a difference for themselves and for others who may be born with this condition in the future,” said Pamela Gavin, NORD’s Chief Strategy Officer. “The project also underscores the important contributions multi-stakeholder research collaborations can make to tackling novel frameworks, and NORD is grateful to those who volunteered to participate in our MLD advisory councils. Our hope is that the success of this study will support the efficient development of treatments and cures for MLD and progress our use of external control arms for the benefit of the entire rare disease community.”
You can drive research forward for #leukodystrophy. Enroll today.
This study is sponsored by NORD with support from FDA CBER FDABAA-19-00123-A3/BAA PMWP#109.