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
  • Nathis-SMA: Prospective study of the natural history of patients with type 2 and 3 Spinal Muscular Atrophy
  • Registry SMA: European Registry of Patients With Infantile-onset Spinal Muscular Atrophy

 

Acronym

AveXis

IONIS-CS11-SHINE

NatHis-SMA

Registry SMA

Study type
RBM RBM RBM-HPS Data collection
Trial type International multicenter trial International multicenter trial International multicenter trial Single-site trial
Intervention AVX-101 ISIS 396443 ISIS 396443
Principal investigator Laurent Servais Laurent Servais Laurent Servais Laurent Servais
Sponsor AveXis BIOGEN AIM AIM
Fundings AveXis BIOGEN Roche / AIM AIM
Study status In preparation Ongoing Ongoing Ongoing
Recruitment status In preparation Closed Closed Ongoing
Population Child Child Adult / Child Child
Contact l.servais@institut-myologie.org l.servais@institut-myologie.org l.servais@institut-myologie.org l.servais@institut-myologie.org
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