After the transplant, her neurologic disease progressed similarly to other patients, but her blood frataxin levels returned to normal and cardiac hypertrophy decreased. This shows that FRDA patients can be treated with bone marrow transplantation, but such treatment alone has no direct effect on progression of neurologic disease.
Tuesday, August 4, 2026
Stem Cell Transplantation in Friedreich Ataxia: Cure for Leukemia but No Effect on Neurological Progression
A. Gitman, N. Bhandari, M. Castellaro, K. Schadt, M. Cancio, and D. R. Lynch, “ Stem Cell Transplantation in Friedreich Ataxia: Cure for Leukemia but No Effect on Neurological Progression,” Annals of Clinical and Translational Neurology (2026): 1–3, doi:10.1002/acn3.70455.
