Mucopolysaccharidosis Type IIIA research hub

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

This study is testing a new gene therapy for young children with Sanfilippo syndrome type A (MPS IIIA), a serious brain condition. It uses the child's own cells, modified to correct the genetic problem. Researchers will check if it's safe and helps improve symptoms.