ItalyResearch and clinical trials
Stem cell transplant in Krabbe disease starting in late childhood: all six children are alive
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 Italy, six patients with Krabbe disease that began late in childhood received a stem cell transplant. Patients were followed for 9 to 19 years. All six patients were alive at the end of follow-up. Four were in nearly normal mental and physical condition. In two patients with widespread brain involvement before the transplant, the outcome was less good.
What does it mean for patients and families?
Transplantation done before symptoms worsen appears to give better results. This is a small group of patients. The transplant decision is made at a specialist center.
Source
Scientific paper: Long-term neurological outcome after hematopoietic stem cell transplant in juvenile Krabbe disease. Journal of Neurology, 2026. DOI: 10.1007/s00415-025-13607-x · PubMed 41604001
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: Krabbe disease (globoid cell leukodystrophy)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.