Published in Gene Therapy Weekly, February 8th, 2001
In its most severe form, the disease, mucopolysaccharidosis 1, causes progressive developmental delays, corneal clouding, lung obstructions, heart disease, severe joint stiffness, and death, usually by age 10. In milder forms, known as Hurler-Scheie syndrome and Scheie syndrome, patients suffer many of the same medical problems, but they develop more slowly, and patients die by...
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