Status | Study |
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: |
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 |
Active, not recruiting |
Study Name: Clinical Evaluator Outcomes Reliability Study Condition: Dystrophinopathies Date: 2014-05-14 |
Recruiting |
Study Name: Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies Condition: Dystrophinopathies, Muscular Dystrophies Date: 2014-04-02 Interventions: Other: blood sample dosage of miRNA |
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- |
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 |
Completed |
Study Name: Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids Condition: Dystrophinopathy Duchenne Muscular Dystrophy Date: 2011-07-05 Interventions: Drug: L-arginine Subjects will receive oral L-Arginine (0.3 grams/kg/day, divided 2 times per day, not t |
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 |