Leukodystrophy Center News

Children’s Hospital of Philadelphia (CHOP) today announced the establishment of the Elise’s Corner Endowed Chair in Alexander Disease Research. Dr. Amy T. Waldman, Medical Director of the Leukodystrophy Center and a pediatric neurologist at CHOP, has been named the inaugural chair holder. Funded by Elise’s Corner and Jim and Marguerite Pearson, the role will support research and treatments for Alexander disease, a form of leukodystrophy that progressively damages the nervous system.

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). 

While unable to speak, patients can understand parents and caregivers, paving the way for future clinical trial design

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

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.

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

Amy Waldman, MD, and a patient family talk about research and fundraising efforts underway that seek to identify better treatment options for Alexander disease.

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.

The Carr-Kefalases and Hammonds presented a check of $110,000 to CHOP’s Leukodystrophy Center.

Adeline Vanderver, MD, aims to take leukodystrophy gene discovery, therapies, support and advocacy to the next level.