Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

Inclusion Criteria:

• Participants must have confirmed diagnosis of infantile-onset Pompe disease definedas: the presence of 2 lysosomal acid α-glucosidase (GAA) pathogenic variants and adocumented GAA deficiency from blood, skin, or muscle tissue; or the presence of 1GAA pathogenic variant and a documented GAA deficiency from blood, skin and muscletissue in 2 separate samples (from either 2 different tissues or from the sametissue but at 2 different sampling dates).

• Participants must have established cross-reactive immunological material (CRIM)status available prior to enrollment.

• Participants must have cardiomyopathy at the time of diagnosis: ie, left ventricularmass index (LVMI) equivalent to mean age specific LVMI

• +1 standard deviation for participants diagnosed by newborn screening orsibling screening;

• +2 standard deviation for participants diagnosed by clinical evaluation.

• Parents or legally authorized representative(s) must be capable of giving signedinformed consent.

Exclusion Criteria:

• Participants with symptoms of respiratory insufficiency, including any ventilationuse (invasive or noninvasive) at the time of enrollment.

• Participants with major congenital abnormality.

• Participants with clinically significant organic disease (with the exception ofsymptoms relating to Pompe disease).

• Participant received any Pompe disease specific treatment, eg enzyme-replacementgene therapy (ERT).

• Participant who has previously been treated in any clinical trial of avalglucosidasealfa.

• Participant not suitable for participation, whatever the reason, as judged by theInvestigator, including medical or clinical conditions, or participants potentiallyat risk of noncompliance to study procedures.

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