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Gene therapy improves bilirubin levels in Crigler-Najjar syndrome

1. In this randomized controlled trial, a high dose of GNT0003 gene therapy was associated with decreased serum bilirubin and need for phototherapy. 2. The most common adverse events associated with GNT0003 were headache and liver enzyme elevations. Evidence Rating Level: 1 (Excellent) Study Rundown: Crigler-Najjar syndrome is a rare recessive disorder resulting in loss

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