TürkiyeResearch and clinical trials
Stem cell transplantation in 17 children with MPS VI in Antalya: all engrafted, enzyme levels normalized
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
Results of stem cell transplantation in children diagnosed with MPS VI at a center in Antalya have been published. The study reviewed the records of 17 children who underwent transplantation in 2021-2023. The transplant took hold in all children, and enzyme levels reached the normal range. In all children who could be measured, the 6-minute walk test improved at a median follow-up of 9 months. The authors argue that transplantation could be a permanent and cheaper alternative to enzyme therapy.
What does it mean for patients and families?
The study is single-center, and the follow-up period is short. Transplantation also carries serious risks; the question of enzyme therapy or transplantation should be discussed together by the family and the metabolic and transplant teams.
Source
Scientific paper: Hematopoietic stem cell transplantation in pediatric patients with type VI mucopolysaccharidosis. Clinical and Experimental Pediatrics, 2025. DOI: 10.3345/cep.2024.02033 · PubMed 40083103
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Maroteaux-Lamy syndrome (MPS VI)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.