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14Cases

2020-08-15 - 2035-12-31

Phase III

Active
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
  • Condition/Disease

    Spinal Muscular Atrophy

  • Test Drug

    AVXS-101

Participate Sites
1Sites

Recruiting1Sites

2020-04-01 - 2026-09-30

Phase II/III

Completed
  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

2021-04-01 - 2027-01-30

Phase III

Active
  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

2024-04-01 - 2033-09-11

Phase III

Active
  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

2022-03-01 - 2024-09-30

Phase III

Completed
  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

2021-09-30 - 2023-08-01

Phase III

Completed
A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA)
  • Condition/Disease

    Pediatric patients with spinal muscular atrophy (SMA)

  • Test Drug

    OAV101

Participate Sites
2Sites

Recruiting1Sites

Study ended1Sites

2023-01-10 - 2031-02-26

Phase IV

Active
  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

2023-12-01 - 2028-06-30

Phase III

Active
  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

1 2