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Archivist
Gene therapy for spinal muscular atropy

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Gene therapy has been an exciting development; once a theoretical dream, it is now, reality. The Archivist has been following the progress of this intervention and now seen patients who have received this ‘miraculous’ therapy. We know that spinal muscular atrophy (type 1) (SMA) is a devastating lethal motor neuron disease where the need for permanent ventilation is expected by age 2 years. Day JW et al. [Lancet Neurol 2021;20:284–93] have reported the findings the of STR1VE, an open label, single arm phase-3 study, recruiting 22 patients with infantile onset SMA. The authors evaluated the safety and efficacy …

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  • Provenance and peer review Commissioned; internally peer reviewed.

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