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TreatSMA vigil on the decisive day

  • 23 October 2018

Today the entire SMA community is awaiting the outcome of the Committee meeting which is taking place at the National Institute of Health and Care Excellence in Manchester. The Committee is to decide whether spinal muscular atrophy will be treated on the NHS.

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Avexis files for regulatory approval of gene therapy

  • 18 October 2018

Avexis has informed today that the company has submitted applications for regulatory approval of AVXS-101, the groundbreaking gene therapy product developed to treat spinal muscular atrophy.

Submissions have been filed with the European Medicines Agency, the US Food and Drug Administration, and the Japanese regulator.

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