an innovative treatment for Crigler-Najjar syndrome

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Health: an innovative treatment for Crigler-Najjar syndrome

An Italian woman with Crigler-Najjar syndrome saw her life change thanks to a revolutionary treatment. She volunteered three years ago to test this gene therapy against this rare disease. – (Franceinfo)

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franceinfo – R.Schapira, C.Napoli, S.Caracciolo, M.Huguet, V.Parent

France Televisions

An Italian woman with Crigler-Najjar syndrome saw her life change thanks to a revolutionary treatment. She volunteered three years ago to test this gene therapy against this rare disease.

For three years, Stefania Moro, 30, has enjoyed a life made up of simple pleasures, like that of driving a car in complete freedom. Since her birth, the young woman has taken heavy treatment to limit the effects of Crigler-Najjar syndrome, from which she suffers. This rare liver disease causes intense jaundice and potential serious neurological risks.

Finally being able to project yourself into the future

In addition to the drug treatment, Stefania Moro had to spend every night completely naked under UV lamps installed in her room. Three years ago, she volunteered to try a revolutionary treatment. It is a gene therapy that acts on bilirubin, this pigment that his liver does not eliminate, which turns his skin yellow and threatens to attack his brain. The results were spectacular. In a few days, the young woman regained a normal complexion, and was finally able to look forward to the future with her partner. They are even expecting a child.


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