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 provides community update on plans for AVXS-101 in Europe

    community update on plans for AVXS-101 in EuropeAveXis has provided SMA Europe with a community update on plans for AVXS-101 in Europe. AVXS-101 is a gene therapy product for SMA.

    STR1VE EU

    • OVERVIEW: STR1VE EU is expected to start enrolling patients in the first half of 2018 in patients with SMA Type 1, studied at multiple centres across the European Union.
    • ADMINISTRATION: In STR1VE EU, AVXS-101 is administered through a one-time IV infusion.
    • WHO: STR1VE EU will enroll approximately 30 patients with SMA Type 1 who are less than six months of age at the time of gene therapy.

    SPRINT

    • OVERVIEW: SPRINT is expected to start enrolling patients in the first half of 2018 in presymptomatic patients with SMA Types 1, 2 and 3.
    • ADMINISTRATION: In SPRINT, AVXS-101 is administered through a one-time IV infusion.
    • WHO: SPRINT is expected to enroll approximately 44 patients with two, three and four copies of SMN2 who are less than six weeks of age and pre-symptomatic at the time of gene therapy.

    REACH

    • OVERVIEW: REACH is expected to start enrolling patients late in 2018 or early in 2019 in
      patients with SMA Types 1, 2 and 3.
    • ADMINISTRATION: In REACH, AVXS-101 is administered through a one-time IT injection. Data from STRONG (the first study of AVXS-101 delivered through IT injection) will help determine the final study design.
    • WHO: REACH is expected to enroll approximately 50 patients with SMA Types 1, 2 and 3 who are between approximately six months and 18 years of age.

    For further information