For Referring Physicians

The RESPOND Study is evaluating potential treatment options for SMA


The study is a phase 4 interventional clinical trial to evaluate the efficacy and safety of nusinersen in participants who previously received onasemnogene abeparvovec. The study will be conducted at approximately 20 sites globally among children three years of age and under.

The investigational drug, nusinersen, is an antisense oligonucleotide, a type of therapy that targets a specific gene and is believed to increase its protein production.


The study is an open-label, single-arm evaluation of nusinersen administered to approximately 60 participants who did not respond optimally to treatment with onasemnogene abeparvovec.

This clinical research study aims to study how children respond when they receive nusinersen following onasemnogene abeparvovec treatment.

Study Duration:
115 weeks (approximately 29 months)

Three-week screening period

95-week treatment period

Three loading doses of nusinersen administered by IT injection at 14-day intervals, a fourth loading dose approximately 30 days after the third dose, and maintenance doses every four months thereafter

17-week follow-up period

Please see for eligibility criteria. Please note: Any patients under your primary care will remain so while they participate in the study.

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