Services to deliver Onasemnogene abeparvovec/Zolgensma

Spinal muscular atrophy, or SMA, is a rare genetic disorder that causes muscle weakness and progressive loss of movement. It is most commonly caused by defects in the gene SMN1. Onasemnogene abeparvovec (Zolgensma, AveXis) is a single-use gene replacement therapy made of a viral vector that has been modified to contain the primary gene for the human survival motor neuron (SMN) protein, which is lacking or mutated in people with SMA. When injected, the vector is expected to carry the gene into the nerve cells, enabl...