Zolgensma onasemnogene abeparvovec

The single highest-yield commercial action: deploy peer mentors at the insurance-denial moment, where access collapse is most acute and family resolve is most fragile.

• Early treatment is the dominant efficacy narrative: families and clinicians alike report dramatically better motor outcomes when Zolgensma is given in the first months of life before symptom progression.
• Cost and global access are the loudest unmet need, with families in low- and middle-income countries describing complete treatment blockades while U.S. families navigate complex prior-authorization and appeals processes.
• Liver-enzyme monitoring and mandatory corticosteroid regimens generate real caregiver burden post-infusion, though parents consistently describe this as manageable relative to the disease outcome it prevents.
• Suboptimal Zolgensma responders are an emerging and commercially significant population, with salanersen trial data showing that some children continue to need add-on or sequential SMN-targeting therapy years after gene therapy.