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Children’s Hospital of Philadelphia Preclinical Study Demonstrates Improved Gene Therapy Approach to Treating Metachromatic Leukodystrophy
In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased effectiveness and safety for the treatment for metachromatic leukodystrophy (MLD), a rare disease in young children characterized by the deficient activity of a critical enzyme. Without effective treatment, MLD leads to rapidly declining neurological skills in young children and is potentially fatal. The promising findings were published in the journal Molecular Therapy Nucleic Acids.
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 Study Highlights Deeper Understanding of Genetic Links between Childhood Speech and Language Disorders
Researchers at Children’s Hospital of Philadelphia (CHOP) announced the results of a new study highlighting significant genetic links in speech and language disorders in children.
Children’s Hospital of Philadelphia Researchers Use AI-Powered Method to Identify Genetic Epilepsies Much Earlier than Current Genetic Diagnosis
New method, bolstered by a study of 89 million points of data, could help patients receive precision care months or years earlier
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
Air Quality FAQs for Patients & Families
You might be feeling anxious about the poor air quality in our region. While this problem is real, it does not mean that there is nothing we can do to help keep our family safe.
CHOP Researchers Develop New Strategy for Assessing Natural History of Leukodystrophies
The methodology developed in this study could help guide clinical trial design for many rare diseases
Hypomelanotic Macules: Clue to Tuberous Sclerosis Complex Diagnosis
Were episodes of a baby suddenly lifting her arms above her head and crying out benign neonatal movements or seizures? How to make the diagnosis.
When Spinal Surgery Can Improve Mobility for Children with Spastic Diplegic Cerebral Palsy
A child’s ability to walk improved because of a partnership between CHOP’s Advanced Tone Management Clinic and Neurosurgery.
Pediatric Reflections: Is Your Nearest ED Prepared to Treat Children?
Pediatric Reflections: Is Your Nearest ED Prepared to Treat Children?