GlobalResearch and clinical trials
Stem cell transplant in 21 children with alpha-mannosidosis: no deaths, some findings decreased
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
In an international study that included Ankara, 21 children who had a stem cell transplant for alpha-mannosidosis were studied. No patient died during follow-up. Liver enlargement, frequent infections, and hearing problems decreased. However, delay persisted in most of the 13 children whose development was tracked. Nine patients had severe infections.
What does it mean for patients and families?
Stem cell transplantation can improve some findings but carries risks. The decision is made at a specialist center together with the family.
Source
Scientific paper: Outcome of Haemopoietic Stem Cell Transplantation in 21 Patients With Alpha-Mannosidosis. Journal of Inherited Metabolic Disease, 2025. DOI: 10.1002/jimd.70047 · PubMed 40551549
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: Alpha-mannosidosis
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.