Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

Inclusion criteria:

• Subject's parents or legal guardians must provide written informed consent prior toany study-related procedures.
• Documented onset of Pompe disease symptoms up to 12 months of age (corrected forgestation if born before 40 weeks); diagnosis of Pompe disease confirmed by acidalpha-glucosidase enzyme deficiency from any tissue source and acid alpha-glucosidasegene mutations.
• Age 0-12 months at enrollment, defined as at the time of providing written informedconsent.
• Cardiomyopathy (abnormal left ventricular mass indices [LVMIs], measured byechocardiography, abnormal value is defined as ≥65 g/m2 for patients up to 12 monthsold) confirmed by cardiologist at study site.

Exclusion criteria:

• Patient who has previously been treated with acid alpha-glucosidase.
• Patient who is participating in another clinical study using any investigationaltherapy.
• Conditions/situations such as:
• Clinical signs of cardiac failure with ejection fraction < 40%.
• Respiratory insufficiency (oxygen saturation < 90% or carbon dioxide partial pressure > 55 mm Hg [venous] or > 40 mm hydrargyrum [arterial] in room air or any ventilatoruse).
• Patients who are dependent on invasive or non-invasive ventilator support.
• Patients with major congenital anomaly or clinically significant intercurrent organicdisease unrelated to Pompe disease.
• Patients not suitable for participation, whatever the reason, as judged by theInvestigator, including medical or clinical conditions, or patients potentially atrisk of noncompliance to study procedures. The above information is not intended to contain all considerations relevant to a patient'spotential participation in a clinical trial.