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11Cases

2020-05-01 - 2026-12-31

Phase II

Active
AN OPEN LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY
  • Condition/Disease

    spinal muscular atrophy

  • Test Drug

    Risdiplam

Participate Sites
1Sites

Recruiting1Sites

2020-10-30 - 2039-12-04

Phase III

Active
A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
  • Condition/Disease

    Duchenne Muscular Dystrophy

  • Test Drug

    PF-06939926

Participate Sites
3Sites

Recruiting3Sites

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

2023-01-10 - 2031-02-26

Phase IV

Active
  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

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

2021-04-01 - 2027-01-30

Phase III

  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

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