What Is SMA Type 1 and How Is It Treated?
SMA type 1 is a severe inherited neuromuscular disease that causes rapid loss of motor neurons, leading to profound muscle…
Zolgensma
A one-time AAV9-delivered gene replacement therapy (onasemnogene abeparvovec) for treating certain infants and young children with spinal muscular atrophy by providing a functional SMN1 gene.