TREATMENT OF POMPE'S DISEASE

摘要

PROBLEM TO BE SOLVED: To provide methods of treating Pompe's disease.SOLUTION: The invention provides methods of treating Pompe's disease using human acid α glucosidase. A preferred treatment regime comprises administering more than 10 mg/kg body weight per week to a patient. The administration is preferably intravenous administration. The methods can be used for treating patients with infantile, juvenile or adult Pompe's disease. The human acid α glucosidase is preferably obtained in milk of a nonhuman transgenic mammal, and is preferably predominantly in a 110 kD form.