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 ...

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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 ...

A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the treatment of older patients with spinal muscular atrophy (SMA).

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 ...