News

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. AveXis dosed first pre-symptomatic patient in SPR1NT trial

    AveXis dosed first pre-symptomatic patient in SPR1NT trial.AveXis, the gene therapy company developing a gene therapy to treat SMA, known as AVXS‐101, recently treated the first patient in a new study known as SPR1NT.

    SPR1NT is a multi-national study which will evaluate AVXS-101 in approximately 44 patients less than six weeks old with SMA Types 1, 2 or 3, who have 2, 3 or 4 copies of SMN2 and have not yet shown symptoms of the disease (pre-symptomatic).

    To find out more about SPR1NT, please read AveXis’ community update.

    Additional information