Cognitive and neuroradiological improvement in three patients with attenuated MPS I treated by laronidase

Stem cell transplantation is not appropriate first-line treatment for attenuated phenotypes of mucopolysaccharidosis type I (MPS I). In three patients with attenuated MPSA I treated by laronidase, Patients 2 and 3 displayed significant cognitive improvement within 2 years; Patients 1 and 3 displayed improvement on MRI scans of the brain.