Spinal Muscular Atrophies-1

  • AveXis: Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
  • IONIS-CS11-SHINE: An Open-label extension study for patients with spinal muscular atrophy who previously participated in investigational studies of ISIS 396443
  • Registry SMA: European Registry of Patients With Infantile-onset Spinal Muscular Atrophy





Registry SMA

Study type
RBM RBM Data collection
Trial type International multicenter trial International multicenter trial Single-site trial
Intervention AVX-101 ISIS 396443 ISIS 396443
Principal investigator Odile Boespflug-Tanguy Andreea Seferian Andreea Seferian
Sponsor AveXis BIOGEN AIM
Fundings AveXis BIOGEN AIM
Study status Ongoing Ongoing In preparation
Recruitment status Ongoing Closed In preparation
Population Child Child Child
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