Existing Class of Drugs May Improve Neurological Function in Patients with Rare, Aggressive Genetic Disorder
Published on in CHOP News

A JAK inhibitor helped patients with Aicardi-Goutières Syndrome meet important developmental milestones.
Published on in CHOP News
A JAK inhibitor helped patients with Aicardi-Goutières Syndrome meet important developmental milestones.
Published on in CHOP News
CHOP leads the way in creating a program to drive breakthroughs to advance the care of children with complex neurologic disorders.
Published on in Children's View
Some diseases affect only a small number of people, and yet CHOP’s commitment to researching and treating them is huge.
Published on in CHOP News
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.
Published on in CHOP News
Amy Waldman, MD, and a patient family talk about research and fundraising efforts underway that seek to identify better treatment options for Alexander disease.
Published on in Neurosciences Update
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.
Published on in CHOP News
The Carr-Kefalases and Hammonds presented a check of $110,000 to CHOP’s Leukodystrophy Center.
Published on in Philanthropy Report
Leukodystrophy Center of Excellence was established in 2015 to address the medically complex needs of children living with leukodystrophies.
Published on in CHOP News
Adeline Vanderver, MD, aims to take leukodystrophy gene discovery, therapies, support and advocacy to the next level.
Published on in CHOP News
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.