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.

Keywords

• Gene therapy
• Liver transplantation
• Phenobarbital
• Phototherapy