Page last updated: May 21, 2026
Research Project Information
Cure Sanfilippo Foundation is funding search to explore re-positioning FDA-approved compounds using cell-based, high-content screening in Sanfilippo Syndrome (MPS III) cell models.
Awardee: Diego Medina, PhD- Telethon Institute of Genetics and Medicine (TIGEM)- Italy
Start Date: June 2018
Updates on Research Project
2024
In 2024, a case report, “Initiation of fluoxetine in a pediatric patient with Mucopolysaccharidosis IIIA: Early observations,” was published in Molecular Genetics and Metabolism Reports.
2022
The primary publication, “Fluoxetine ameliorates mucopolysaccharidosis type IIIA” was published in Molecular Therapy in 2022 regarding the cell-based drug screening results and Sanfilippo A mouse study.
October 2019
In October 2019, TIGEM presented preliminary results of this study at the Conference of Telethon Fundamental Associations in Italy. Read more.
