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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, Penn Medicine Researchers Report High-Throughput Variant Re-Classification for Alagille Syndrome Disease Gene
Findings will help provide more accurate diagnoses to families.
Q&A with Binita M. Kamath, MBBChir, MRCP, MTR
CHOP’s new Chief of the Division of Gastroenterology, Hepatology and Nutrition shares her vision for the department’s future.
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
The Latest in Liver Research: 2022
Active research at the Fred and Suzanne Biesecker Pediatric Liver Center.
Liver Disease in Children
Research in the lab and at the bedside offer new hope for young patients with biliary atresia, Alagille syndrome and other chronic liver disorders.
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.