Orphanet Journal of Rare Diseases (Mar 2024)
Crigler-Najjar syndrome: looking to the future does not make us forget the present
Abstract
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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