Positive preliminary results from SMA gene therapy trial

Published Date
Beatriz Bustillo Ramirez
Orange box stating: Breaking news research

AveXis has shared preliminary data from its phase 3 clinical trial (STR1VE) in spinal muscular atrophy type 1.

The treatment, Zolgensma, is a gene therapy designed to replace the faulty gene that causes the condition. The trial results so far show that children treated with Zolgensma are living longer and achieving significant development milestones.

For more information read AveXis’ press release.

If you have any questions about this news story or any other SMA research, please contact the MDUK Research Line on 020 7803 4813 or email research@musculardystrophyuk.org.

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