Join a community of families living with leukodystrophy
Find courage, strength, and hope in the stories of amazing families
Who we are...
We are a community of people united by our love for a child,
grandchild, sibling, friend, or student affected by the 50 forms of leukodystrophy. With the support and guidance of nurses, doctors, researchers, social workers, child life specialists and other patient advocates, we have created a resource for people caring for a loved one affected by leukodystrophy.
We have tried to address the most frequently asked questions and common issues shared by our community. Experts will keep updating this page with the best information and research.
We will provide links to doctors, hospitals, articles, foundations, and programs that will support you and your family. We are funded through the generous support of the Calliope Joy Foundation.
Learn about world-renowned specialists, Leukodystrophy Centers of Excellence, and groundbreaking research.
Network with other families and leading specialists.
Patient and Parent Advocates
Meet other patient advocates and share stories with others fighting to defeat leukodystrophy. Connect with others interested in funding research, championing newborn screening, and advocating for improved care and treatments.
We are so pleased to announce that Takeda has announced a US trial for their enzyme replacement therapy to treat metachromatic leukodystrophy patients. This clinical trial represents a "game-changing" moment for leukodystrophy patients.
If you would like to learn more to see if your child can participate, please go to this link. Lurie Children's Hospital's Leukodystrophy Program is overseeing the first site in Chicago, IL.
Embolden Study - They are looking for children 18 months to six years old to participate in the clinical study.