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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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Roche announces positive interim results of risdiplam trials

  • 3 October 2018

Roche today announced interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam as a treatment for spinal muscular atrophy. The data were presented yesterday at the 23rd International Annual Congress of the World Muscle Society in Mendoza, Argentina.

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