First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.
SMA is a genetic disorder that can cause rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing, and basic movement.
