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NEW YORK (AP) — Shares of BioMarin Pharmaceuticals Inc. jumped to an all-time high Monday after the company said an experimental treatment for a rare genetic disease called Morquio A Syndrome succeeded in a late-stage clinical trial.
BioMarin said patients who were treated with its drug GALNS, or BMN-110, had a larger improvement in the distance they could walk in six minutes. It said patients who took the drug for 24 weeks also had greater stair-climbing speed, greater improvements in lung function, and lower levels of a type of carbohydrate in their urine. BioMarin expects to file for marketing approval of GALNS in the first quarter of 2013.
Shares of BioMarin rose $11.09, or 30 percent, to $48.50 in afternoon trading. Earlier the stock peaked at $49.52, an all-time high.
Morquio A Syndrome, or mucopolysaccharidosis IV-A, is a genetic disease in which the body does not produce enough of the GALNS enzyme, and a compound called keratan sulfate builds up in organs and tissues. Morquio A Syndrome causes skeletal and joint problems and shortness, and can also cause hearing loss, clouded vision and heart valve disease.
BioMarin makes two drugs that treat other types of the disease: Naglazyme, a treatment for mucopolysaccharidosis VI, and Aldurazyme, a treatment for mucopolysaccharidosis I.