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Page last checked: 22nd November 2022
Last updated: April 2021


In the Phase 3 STR1VE trial with 33 infants aged 6 months or under with SMA Type 1 and with one or two SMN2 copies, Zolgenmsa™ treatment was confirmed to result in prolonged event-free survival, improvements in muscle function, and achievement of key milestones.

Previous Updates

27th September: Great Ormond Street Hospital is actively screening infants; The John Walton Muscular Dystrophy Centre for Research in Newcastle will be activated very soon.

9th February: STR1VE EU, Phase 3 pivotal trial announced to begin in the first half of 2018. The study is a single-arm trial, meaning that there is no patient group receiving a placebo; rather, the trial will compare the motor milestone achievements of patients receiving AVXS-101 to natural history information from untreated SMA patients.