Haploidentical haematopoietic stem cell transplantation combined with post-transplant cyclophosphamide in neuronal ceroid lipofuscinosis: Experience in eight patients

CONCLUSION: This study demonstrated that it is safe and efficacious to treat NCLs with haplo-HSCT. Transplantation should be performed at an early stage for the survival quality of pediatric patients.PMID:38044188 | DOI:10.1016/j.medcli.2023.07.029

Source: Medicina Clinica - December 3, 2023 Category: General Medicine Authors: Zeliang Song Xiaodai Cui Zhaoxia Zhang Rong Liu Xiaodong Shi Source Type: research