Treatment of pediatric patients with Sanfilippo syndrome (MPS IIIA and IIIB) with unrelated umbilical cord blood transplantation

Umbilical cord blood transplantation (UCBT) favorably alters the natural history of young pediatric patients with Hurler syndrome (MPS I). We asked whether it would also be effective for patients with Sanfilippo syndrome (MPS III). Patients with Sanfilippo syndrome have less somatic and more CNS disease than patients with other MPS syndromes. They are typically diagnosed between 3 and 5 years of age. Without treatment, they develop severe neurologic dysfunction, deafness, inability to speak or ambulate, and severely autistic behaviors.