Mucopolysaccharidosis is a metabolic disorder caused by missing or deficient enzymes needed to break down the keratan sulfate sugar chain. It is physically manifested through skeletal abnormalities, such as a flattening or curvature of the spine and dysplasia of the hips and knees.
Vimizim was also granted a priority review designation, a designation given to drugs that offer major treatment advances, according to the report.
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