Spinal muscular atrophy long-term outcome data reshapes treatment expectations

Spinal muscular atrophy was a no-effective-therapy category until 2016. Nusinersen, onasemnogene abeparvovec, and risdiplam have transformed outcomes for newborn-screened and presymptomatic infants and provide meaningful benefit in symptomatic patients. The next-cycle conversation is multi-year outcome data, combination strategies (gene therapy plus SMN-modifier), and the question of whether late-treated patients converge with early-treated patients over time. The newborn screening rollout is the addressable-population enabler.