Crigler-Najjar: gene therapy shows its effectiveness

« Immunologie et Thérapie Génique des Maladies du Foie » de Généthon, dirigée par le Dr Giuseppe Ronzitti
Results published in The New England Journal of Medicine demonstrate the safety and tolerance for gene therapy in Crigler-Najjar syndrome as well as its efficacy at the highest dose. This rare liver disease is characterized by a toxic build-up of bilirubin in the body, which can cause significant neurological damage and become fatal.

Spending 12 hours a day under phototherapy to keep bilirubin levels below the toxicity threshold and avoid major neurological damage: this, along with liver transplants when it’s possible, is the only treatment available for people suffering from Crigler-Najjar syndrome.

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