Basel, October 1, 2020 – Novartis Gene Therapies today announced new interim data from the ongoing Phase 3 STR1VE-EU clinical trial for Zolgensma® (onasemnogene abeparvovec) that demonstrated patients with spinal muscular atrophy (SMA) Type 1 continued to experience significant therapeutic benefit, including event-free survival, rapid and sustained improvement in motor function and motor milestone achievement, including for some patients with more aggressive disease at baseline compared to previous trials. SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene that results in the progressive and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing, and basic movement.1,2,3 These data as of December 31, 2019, and presented today during a virtual Clinical Trial Poster Session as part of the World Muscle Society (WMS) 2020 Virtual Congress, support the robust clinical evidence that has demonstrated a consistent, transformative benefit across Zolgensma clinical trials for the treatment of patients with SMA.
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Zolgensma® data including patients with more severe SMA at baseline further demonstrate therapeutic benefit, including prolonged event-free survival,...
