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CHOP Named Qualified Treatment Center for Lenmeldy, the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy
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).
Children’s Hospital of Philadelphia 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
Existing Class of Drugs May Improve Neurological Function in Patients with Rare, Aggressive Genetic Disorder
A JAK inhibitor helped patients with Aicardi-Goutières Syndrome meet important developmental milestones.
None Too Rare
Some diseases affect only a small number of people, and yet CHOP’s commitment to researching and treating them is huge.
Leukodystrophy Patient Featured on Seeking Solutions with Suzanne
Elise has Alexander disease, a type of leukodystrophy. Watch the video to learn how the Leukodystrophy Center at CHOP is committed to helping kids like Elise.
Washington Post Highlights Alexander Disease Research Efforts
Amy Waldman, MD, and a patient family talk about research and fundraising efforts underway that seek to identify better treatment options for Alexander disease.
Advancing Understanding and Care of Leukodystrophies
The Leukodystrophy Center sees patients from all over the country and the world. Our services include a newborn diagnostic clinic and a comprehensive care clinic that provides state-of-the art care.
Families Affected by Leukodystrophy Donate $110,000 to Children's Hospital of Philadelphia
The Carr-Kefalases and Hammonds presented a check of $110,000 to CHOP’s Leukodystrophy Center.
Meet the New Program Director of the Leukodystrophy Center of Excellence
Adeline Vanderver, MD, aims to take leukodystrophy gene discovery, therapies, support and advocacy to the next level.
2016 Black Tie Tailgate Raises More than $550,000 for the Division of Neurology at CHOP
The 2016 Philadelphia Auto Show’s Black Tie Tailgate broke records, with more than 4,800 people helping to raise over $550,000 for the Division of Neurology.