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Orphanet Journal of Rare Diseases
Published in: Orphanet Journal of Rare Diseases 1/2024

Open Access 01-12-2024 | Phototherapy | Letter to the Editor

Crigler-Najjar syndrome: looking to the future does not make us forget the present

Authors: Fabiola Di Dato, Giuseppe D’Uonno, Raffaele Iorio

Published in: Orphanet Journal of Rare Diseases | Issue 1/2024

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Abstract

Recently, the safety and efficacy of gene therapy were evaluated in patients with Crigler-Najjar syndrome (CNS). Although it is a promising curative option for CNS, many doubts still persist about its long-term efficacy and safety. Furthermore, there is a risk of overlooking several unresolved problems still present in current clinical practice. This letter is a call for action on crucial open issues that remain nowadays an unmet need in the management of CNS patients.
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Metadata
Title
Crigler-Najjar syndrome: looking to the future does not make us forget the present
Authors
Fabiola Di Dato
Giuseppe D’Uonno
Raffaele Iorio
Publication date
01-12-2024

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