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An Open-Label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in Naive and Alglucosidase Alfa Treated Late-Onset Pompe Disease Patients
Men and women who are at least 18 years old and have been diagnosed with late-onset Pompe disease are needed to participate in a research study for an investigational drug. Patients may participate if they have never received enzyme replacement therapy, and they may also participate if they have been receiving alglucosidase alfa for at least 9 months. Interested patients must also be able to walk at least 50 meters without stopping and without the use of an assistive device. (REF# 47178)