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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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TreatSMA met with the Roche UK team

  • 3 October 2018

At the request from TreatSMA, on Monday 24 September five members of TreatSMA held a meeting with Roche in London. We discussed our advocacy work and general wider issues facing the SMA Community.

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