1.
Orphanet J Rare Dis
; 19(1): 102, 2024 Mar 07.
Artículo
en Inglés
| MEDLINE
| ID: mdl-38448957
RESUMEN
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.