This study focuses on children under 2 years of age who have Spinal Muscular Atrophy (SMA), a genetic condition that affects the nerves controlling muscle movement, causing muscle weakness and movement difficulties. The study will test a medicine called apitegromab, given through intravenous infusion (delivered directly into a vein).
The purpose of this research is to understand how apitegromab works in young children with SMA and to evaluate its effects on their movement abilities. All children in the study must already be receiving or have received other approved treatments for SMA, such as onasemnogene abeparvovec-xioi, nusinersen, or risdiplam.
The study will last for 48 weeks, during which children will receive regular infusions of the study medication. Throughout the study, doctors will monitor how the medicine moves through the body and its effects on muscle function. This is a double-blind study, which means neither the participants nor the doctors directly involved in their care will know which treatment each child receives.
Belgium 
France 
Italy 
Spain 
The Netherlands 
