Harlow is defying the odds after receiving groundbreaking gene therapy for a rare brain disorder. Her progress has been called "a miracle" by her family ...

The Food and Drug Administration on Monday approved a gene therapy for metachromatic leukodystrophy, a devastating genetic disorder that eats away at affected children’s neurons. The medicine, ...

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This severe leukodystrophy, caused by galactosylceramidase (GALC) mutations, primarily affects infants and young children, causing symptoms such as irritability, feeding difficulties, seizures, muscle ...

TOKYO and LONDON and BOSTON, March 18, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients ...

PHILADELPHIA, March 11, 2025 /PRNewswire/ -- In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased ...

PHILADELPHIA, April 30, 2025 /PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania announced the first ever ...