Zolgensma® meaningfully improves spinal muscular atrophy in patients
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene…
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.