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Dystrophinopathy - 9 Studies Found

Status Study
Completed Study Name: Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
Condition:
  • Dystrophinopathy
  • Duchenne Muscular Dystrophy
  • Dystrophinopathies,
  • Muscular Dystrophies

Date: 2014-04-02
Interventions: Other: blood sample dosage of miRNA
Active, not recruiting Study Name: Clinical Evaluator Outcomes Reliability Study
Condition: Dystrophinopathies
Date: 2014-05-14
Active, not recruiting Study Name: Study of Ataluren for Previously Treated Patients With nmDBMD in Europe, Israel, Australia, and Canada
Condition:
  • Duchenne Muscular Dystrophy
  • Becker Muscular Dystrophy
  • Date: 2012-03-15
    Interventions: Drug: Ataluren Oral powder for suspension taken 3 times per day (10 mg/kg in the morning, 10 mg/kg at mi
Enrolling by invitation Study Name: Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
Condition:
  • Muscular Dystrophy, Duchenne
  • Muscular Dystrophies
  • Muscular
    Date: 2014-03-17
    Interventions: Drug: Ataluren Other Name: PTC124
Completed Study Name: Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy
Condition:
  • Muscular Dystrophy, Duchenne
  • Muscular Dystrophies
  • Muscular
    Date: 2013-03-26
    Interventions:
    • Drug: Ataluren Ataluren PO 10-
Completed Study Name: Correlation Between Respiratory Impairment and Phonemes Alteration in Dystrophinopathy Patients With Respiratory Failure
Condition: During the Medical Appointment, the Speech Therapist Will Perform Phonetic Tests
Date: 2015-04-03
Interventions: Behavioral: phonetic tests
Enrolling by invitation Study Name: Study of Ataluren for Previously Treated Patients With nmDBMD in the US
Condition:
  • Duchenne Muscular Dystrophy
  • Becker Muscular Dystrophy

Date: 2010-11-22
Interventions: Drug: Ataluren Oral powder for suspension taken 3 times per day (10 mg/kg in the morning, 10 mg/kg at mi
Active, not recruiting Study Name: Study of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
Date: 2016-06-16
Interventions: Drug: Ataluren Ataluren Other Names: