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.
Crigler-Najjar syndrome: looking to the future does not make us forget the present / DI DATO, Fabiola; D'Uonno, Giuseppe; Iorio, Raffaele. - In: ORPHANET JOURNAL OF RARE DISEASES. - ISSN 1750-1172. - 19:(2024). [10.1186/s13023-024-03108-x]
Crigler-Najjar syndrome: looking to the future does not make us forget the present
Fabiola Di DatoPrimo
;Giuseppe D'UonnoSecondo
;Raffaele Iorio
Ultimo
2024
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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
