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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.

March 11, 2025


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