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Crigler-Najjar syndrome: looking to the future does not make us forget the present
Fabiola Di Dato1, Giuseppe D'Uonno1, Raffaele Iorio2
1Department of Translational Medical Science, Section of Pediatrics, University of Naples Federico II, Via Sergio Pansini 5, 80131, Naples, Italy.
None:
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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