Leukodystrophy Center News

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.

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Children’s Hospital of Philadelphia Researchers Find Some Patients with Aicardi Goutières Syndrome Have Normal IQ

Jun 24, 2024

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

Sep 10, 2020

A JAK inhibitor helped patients with Aicardi-Goutières Syndrome meet important developmental milestones.

Leukodystrophy Patient Featured on Seeking Solutions with Suzanne

Sep 24, 2018

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.

None Too Rare

Oct 23, 2018

Some diseases affect only a small number of people, and yet CHOP’s commitment to researching and treating them is huge.

Washington Post Highlights Alexander Disease Research Efforts

Apr 23, 2018

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

Feb 20, 2018

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

Feb 1, 2018

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

Nov 23, 2016

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

Feb 19, 2016

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.

CHOP Launches Leukodystrophy Center of Excellence for Children With Degenerative Inherited White Matter Diseases

May 18, 2015

The New Leukodystrophy Center of Excellence will offer multidisciplinary diagnostic and clinical services for children with degenerative inherited white matter diseases, and be dedicated to advancing research for more effective treatments.

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