Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

The total study duration per patient will be 4 to 9 weeks that consist of a screening period
(from 2 days to 4 weeks), treatment visit (1 day), and a follow up call (≥30 days).

Inclusion Criteria:

A patient must meet all of the following criteria to be eligible for this study:

- The patient and/or the patient's parent/legal guardian is willing and able to provide
signed informed consent.

- The patient has a confirmed GAA enzyme deficiency from skin, blood, or muscle tissue
and/or 2 confirmed GAA gene mutations.

- Infant and toddler Pompe disease patients can be included in the study only under
condition (minimal body weight) that the trial-related blood loss (including any
losses in the maneuver) will not exceed 3 percent of the total blood volume during a
period of 4 weeks and will not exceed 1 percent at any single time.

- The patient, if female and of childbearing potential, must have a negative pregnancy
test (urine beta-human chorionic gonadotropin) at screening. Note: All female patients
of childbearing potential and sexually mature males must agree to use a medically
accepted method of contraception throughout the study.

- For patients previously treated with alglucosidase alfa the patient has received
alglucosidase alfa for at least 6 months.

Exclusion Criteria:

A patient who meets any of the following criteria will be excluded from this study:

- The patient is participating in another clinical study using an investigational
product.

- The patient, in the opinion of the Investigator, is unable to adhere to the
requirements of the study.

The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial.