Leukodystrophy Center

Learning your child has leukodystrophy is challenging. These inherited disorders of the white matter of the brain and spinal cord can cause problems with movement, speaking, vision, hearing and overall development, and they can be difficult to diagnose and manage. The Leukodystrophy Center at Children's Hospital of Philadelphia (CHOP) is here for you. We are one of the most experienced teams when it comes to diagnosing this condition and providing care. Our experts provide comprehensive diagnostic testing and the most advanced treatments available to infants, children, adolescents, and adults. Our team of doctors, clinicians, and researchers are actively involved in ongoing research to better understand leukodystrophies and find new treatments.
How we serve you
At the Leukodystrophy Center, your child's care team provides cutting-edge clinical care to maximize health, quality of life and everyday function. CHOP is also dedicated to advancing clinical care. We are using the power of discovery to improve the diagnosis and treatment of individuals with leukodystrophy.
Conditions we treat
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Leukodystrophy -
4H leukodystrophy -
Adrenoleukodystrophy (ALD) -
Aicardi-Goutieres syndrome (AGS) -
Alexander disease - Canavan disease
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Hypomyelination with atrophy of the basal ganglia and cerebellum (TUBB4A-Associated Disorder) - Krabbe disease (globoid cell leukodystrophy)
- Megalencephalic leukoencephalopathy with subcortical cysts (MLC)
- Metachromatic leukodystrophy (MLD)
- Multiple Sulfatase Deficiency (MSD)
- Pelizaeus-Merzbacher disease (PMD)

Why choose CHOP's Leukodystrophy Center
The Leukodystrophy Center at Children’s Hospital of Philadelphia provides comprehensive clinical care, diagnostic testing, and the most advanced treatments available to infants, children and adolescents with inherited white matter diseases.

Meet your team
The Leukodystrophy Center at CHOP includes a broad team of specialists dedicated to providing care, testing and treatments to infants and children with inherited white matter diseases.

Our research
The Leukodystrophy Center at Children’s Hospital of Philadelphia (CHOP) is doing many studies to improve understanding, diagnosis and treatment of different leukodystrophies. We are always working on new studies. Check back often to stay up-to-date on our progress.

Our resources
We have created resources to help you find answers to your questions and feel confident with the care you are providing your child.

Resources for professionals
Everything you need to support your patient’s health, created and updated by our CHOP community of experts.

Your child’s appointment
Appointments at the Leukodystrophy Center are multi-disciplinary so that patients can see many important team members in one day.
Lenmeldy has the potential to stop or slow disease progression with a single cell-based treatment.
CHOP’s Leukodystrophy Center, one of the leading centers of its kind in the nation, now offers Lenmeldy to treat children with metachromatic leukodystrophy (MLD).
CHOP Researchers Find Some Patients with Aicardi Goutières Syndrome Have Normal IQ
While unable to speak, patients can understand parents and caregivers, paving the way for future clinical trial design.
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