Harada H, Uchiwa H, Nakamura M et al.
Kurume University Medical Center, Kurume, Japan.
Mol Genet Metab 2011;103:215–9.
Editor’s note: This is a single case report of a 49-year-old woman with muco-polysaccharidosis type I (MPS I) and valvular heart disease who received laronidase enzyme replacement therapy (ERT). MPS I is a heterogeneous condition and, in its mildest form, can be associated with a virtually normal lifespan and little or no cognitive impairment. Laronidase ERT has been shown to be safe and effective in animal and human studies, and has been shown to improve respiratory function and physical capacity in patients with MPS I.