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Pictured from left: Dr. Matt Jolley and Dr. Kevin Whitehead with 3D-printed heart models.
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Image-Based Precision Medicine

CHOP’s Cardiac Center harnesses new technologies, such as virtual reality, to deliver individualized, patient-specific care.

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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.

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Solution for Rare and Complex Early-Onset Kidney Stones

This clinical case study describes a patient with early onset cystinuria, a rare genetic cause of kidney stones, that was first managed by multiple unsuccessful procedures and surgeries before referral to Children’s Hospital of Philadelphia.

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Vaccine News & Notes — March 2025

Find info about the latest cases of measles and a video by Dr. Paul Offit providing a generational perspective on vaccines.

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