Spinraza Trial Begins Treating Children Who Failed to Respond to Gene Therapy

Spinraza Trial Begins Treating Children Who Failed to Respond to Gene Therapy

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Spinraza Trial Begins Treating Children Who Failed to Respond to Gene Therapy

The first patient has been dosed in a Phase 4 trial exploring the safety and potential benefits of Spinraza (nusinersen) in children with spinal muscular atrophy (SMA) who failed to respond adequately to Zolgensma. The two-year study, called RESPOND (NCT04488133), was launched last year by Biogen, Spinraza’s developer, and aims to enroll around 60 children, from 3 months to 3 years old, who have the potential to gain additional clinical improvements beyond those provided by…

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