A recent study conducted at the Capital Institute of Pediatrics in Beijing, China, explored the use of haploidentical hematopoietic stem cell transplantation (HSCT) combined with post-transplant cyclophosphamide (PT/Cy) in the treatment of Neuronal Ceroid Lipofuscinoses (NCLs), a rare lysosomal storage disorder. The study involved eight pediatric patients with NCLs who underwent the procedure. The results showed that the treatment was safe and effective, with all patients achieving full donor chimerism and experiencing no severe transplantation-related complications. However, the progression of mental motor disorders, myoclonic seizures, and vision loss continued, albeit at a slower rate, after transplantation. The study concluded that early transplantation is crucial for improving the survival quality of pediatric patients with NCLs. [Extracted from the article] more...