SAFETY AND EFFICACY OF THE STEM CELL TRANSPLANTATION IN FRIEDREICH’S ATAXIA: A REPORT OF THREE CASES

Riza Azeri, Duygu Koyuncu Irmak, Eda Sun, Erdal Karaöz; Int J Physiother. Vol 8(1), 31-35, February (2021) doi:10.15621/ijphy/2021/v8i1/903 

Case Summary: Here, we report three FRDA cases treated with four consecutive allogeneic transplantations of umbilical cord-derived MSCs with 30 days interval, upon per patient regulatory approvals for advanced cellular therapy. Outcome Measures: The cases were followed up after the treatment in means of the therapeutic effect of the cellular treatment by attenuating the neurological findings and gene expression parameters. Conclusions: Closely followed promising safety and efficacy outcomes demonstrated that the MSC treatment for FRDA might positively affect the clinical results caused by the defect in this genetic-based disease.