Leukodystrophy Center Resources

Advocacy Organizations

Find information about adrenoleukodystrophy (ALD), resources for families, upcoming events, and ways to support.

With its mission to improve health outcomes for patients with X-linked adrenoleukodystrophy, ALD Connect provides education and support information for families, research information, ways to get involved, and more.

Chloe’s Fight Rare Disease Foundation is dedicated to supporting the development of cures and treatments for childhood lysosomal storage diseases (LSDs) by funding research, advocating policy for patients, and raising awareness.

The Foundation's mission is to fund research for the benefit of all those who suffer from GM1 gangliosidosis. Their website provides information about GM1, clinical trials, videos, patient stories, ways to get involved and more.

Offers support to families recently diagnosed with or living with metachromatic leukodystrophy (MLD). The website links to relevant clinical and research opportunities available at CHOP.

Elise’s Corner is a community group focused on spreading awareness of Alexander disease and aiding research efforts to find treatments and a cure for this rare genetic disorder.

Provides assistance and support to families affected by leukodystrophy, funds research projects, raises public awareness, and collaborates with international organizations to pool resources to develop research.

La Fundación Lautaro te Necesita es una asociación civil sin fines de lucro que está funcionando desde 2014 en Argentina. Su misión es contribuir a mejorar la calidad de vida de todas aquellas personas afectadas por leucodistrofias.

Gavin Flying For A Cure was created to honor Gavin Quimby (Super Gav) and to bring awareness to metachromatic leukodystrophy (MLD). Read Gavin's story and find out how you can get involved.

Raises awareness and funds for Alexander disease and helps those with this rare disorder and other types of leukodystrophy get the care they need.

Hunter's Hope Foundation is a non-profit organization committed to giving hope through education and awareness, research, and family care for leukodystrophies.

It's committed to pioneering a patient-centered care model and strengthening groundbreaking research by engaging the patients and caregivers with the Krabbe research community, to ensure the needs of Krabbe patients are being voiced.

Find answers to frequently asked questions and common issues shared by the leukodystrophy community. Access disease and research information and links to support resources.

It aims to exponentially accelerate the discovery of novel genetic variations responsible for childhood leukodystrophies and to translate these findings into clinical treatments.

A non-profit organization serving families throughout the world affected by metachromatic leukodystrophy. Find information about MLD, including educational, research, clinical trials, advocacy and more.

The MSD Action Foundation works to promote and support clinical research advancements that will lead to a life-saving treatment or cure for children with MSD.

Find information about Pelizaeus-Merzbacher disease (PMD), ways to communicate with other families, research information into causes, treatment, prevention and cure of PMD, and much more.

Their fundraising efforts helped establish the nation's first Leukodystrophy Center of Excellence at CHOP. Learn about their cupcake challenge and how to get involved, and find information about current and upcoming clinical trials.

The Canavan Foundation is a not-for-profit organization dedicated to educating at-risk populations about Canavan disease and other Jewish genetic diseases and supporting research looking into a cure.

Provides information about H-ABC to spread awareness about this degenerative disease, and raises funds for research on gene therapy treatments that could stop the degenerative effects of H-ABC.

Works to promote the expansion of newborn screening, and to further education, awareness and research of Krabbe disease and cystic fibrosis to provide a better treatment and a cure for children.

The M.O.R.G.A.N. Project is a national, community-based organization of volunteers dedicated to promoting awareness and facilitating support of parents caring for their children/young adults with special healthcare needs.

The Yaya Foundation focuses on finding a cure for 4H leukodystrophy by supporting research, raising awareness, improving education and connecting families.

ULF is a non-profit, voluntary health organization dedicated to funding cutting-edge research and to providing patients and their families with disease information and medical referrals.

Provides information about multiple sulfatase deficiency (MSD), patient resources, events, family stories, research information and more.


Videos

Family from leukodystrophy event

This video includes interviews with families whose children have leukodystrophies, and highlights from the 2018 Leukodystrophy Center Family Conference at CHOP.


Websites

By connecting families and supporting leukodystrophy research, A Rare Ruby has established itself as one of the key resources for individuals and families affected by H-ABC and TUBB4A-related leukodystrophy.

The mission of the National Institute of Neurological Disorders and Stroke (NINDS) is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.

The National Organization for Rare Disorders (NORD) provides advocacy, education and other services to improve the lives of all people affected by rare diseases.

It's an alliance of like-minded organizations interested in the development of therapies and improving the quality of life for those with leukodystrophies.