Infant with rare disease receives customized gene therapy

National Institutes of Health (NIH) | (.gov)·Jun 3

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A baby with a rare disease has received customized gene therapy, reportedly the first personalized treatment of its kind. The infant, who was treated at the Children's Hospital of Philadelphia, has shown significant progress, including taking first steps. The successful treatment is seen as a breakthrough in the development of custom gene therapies for incurable diseases.

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Infant with rare disease receives customized gene therapy

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