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14Cases

2019-06-01 - 2021-08-01

Phase III

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
  • Condition/Disease

    Spinal Muscular Atrophy Type 1

  • Test Drug

    AVXS-101

Participate Sites
1Sites

Recruiting1Sites

2014-07-01 - 2020-12-31

Phase III

  • Condition/Disease

  • Test Drug

Participate Sites
3Sites

Recruiting2Sites

Terminated1Sites

1 2