Spinal muscular atrophy (SMA) is a disorder affecting the motor neurons—nerve cells that control voluntary muscle movement. These cells are located in the spinal cord. Because the muscles cannot ...

Key to that mission is the “STEER” study, a late-stage trial of children with moderate SMA who are older than two years and have never before received certain targeted medicines like Zolgensma, Biogen ...

The pivotal trial enrolled only babies under 6 months. Two-year-olds born with the most common and severe form of SMA would likely have lost too many neurons to benefit from the therapy.

OAV101 IT, a version of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s administered into the spinal canal, can be safely given to people with spinal muscular atrophy (SMA) who were ...

Newly approved SMA therapies show promise but face global accessibility challenges, widening healthcare disparities due to high costs and limited availability. Newborn screening for SMA is ...