Research for SMA

  • AveXis registry: A Prospective, Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA) – (RESTORE).
    • To assess the long-term outcomes of patients with a diagnosis of SMA and also characterize and assess long-term safety and effectiveness of AVXS-101.
    • To assess overall survival of all patients with SMA.
    • No age restriction. Must have genetic confirmation of SMA on or after 24 May 2018.