This clinical trial is focused on studying a rare genetic disorder known as Glycogen Storage Disease Type II, commonly referred to as Pompe Disease. This condition affects the body’s ability to break down glycogen, a stored form of sugar, leading to muscle weakness and other serious health issues. The study is specifically targeting infants with the early-onset form of this disease, known as Infantile-Onset Pompe Disease (IOPD). The treatment being tested is called Avalglucosidase Alfa, which is a type of enzyme replacement therapy. This therapy aims to provide the missing enzyme that helps break down glycogen in the body.

The purpose of the study is to evaluate the effectiveness and safety of Avalglucosidase Alfa in infants who have not previously received treatment for IOPD. Participants in the study will receive the treatment through an intravenous infusion, which means the medication is delivered directly into the bloodstream through a vein. The study will monitor the participants over a period of time to assess their survival and whether they can live without the need for invasive breathing support, such as a ventilator, after 52 weeks of treatment.

Throughout the study, various health indicators will be observed, including changes in heart size, growth measurements like body length and weight, and motor skills development. The study will also track any side effects or reactions to the treatment. The goal is to gather comprehensive data on how Avalglucosidase Alfa impacts the health and development of infants with IOPD, providing valuable insights into the potential benefits and risks of this treatment option.