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19Cases

2020-01-01 - 2025-12-31

Phase III

Completed
A Multicenter, Single Arm, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Satralizumab in Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)
  • Condition/Disease

    Neuromyelitis Optica Spectrum Disorder (NMOSD)

  • Test Drug

    ENSPRYNG 120mg for SC Injection

Participate Sites
4Sites

Recruiting4Sites

2017-09-01 - 2021-12-31

Phase III

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
  • Condition/Disease

    Genetic Disorders – Duchenne Muscular Dystrophy

  • Test Drug

    Ataluren (PTC124)

Participate Sites
2Sites

Recruiting2Sites

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

2021-04-01 - 2027-01-30

Phase III

  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

2020-04-01 - 2026-09-30

Phase II/III

Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
  • Condition/Disease

    Spinal Muscular Atrophy

  • Test Drug

    Nusinersen

Participate Sites
2Sites

Recruiting2Sites

2023-01-10 - 2035-06-15

Phase IV

  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

1 2