9 studies found for:    "Dystrophinopathy"
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"Dystrophinopathy" (9 records)
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Rank Status Study
1 Completed Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
Conditions: Dystrophinopathy;   Duchenne Muscular Dystrophy;   Becker's Muscular Dystrophy
Intervention: Drug: L-arginine
2 Recruiting Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies
Conditions: Dystrophinopathies,;   Muscular Dystrophies
Intervention: Other: blood sample
3 Active, not recruiting Study of Ataluren for Previously Treated Patients With nmDBMD in Europe, Israel, Australia, and Canada
Conditions: Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy;   Dystrophinopathy
Intervention: Drug: Ataluren
4 Enrolling by invitation Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
Conditions: Muscular Dystrophy, Duchenne;   Muscular Dystrophies;   Muscular Disorders, Atrophic;   Muscular Diseases;   Musculoskeletal Diseases;   Neuromuscular Diseases;   Nervous System Diseases;   Genetic Diseases, X-Linked;   Genetic Diseases, Inborn
Intervention: Drug: Ataluren
5 Active, not recruiting Clinical Evaluator Outcomes Reliability Study
Condition: Dystrophinopathies
Intervention:
6 Active, not recruiting Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy
Conditions: Muscular Dystrophy, Duchenne;   Muscular Dystrophies;   Muscular Disorders, Atrophic;   Muscular Diseases;   Musculoskeletal Diseases;   Neuromuscular Diseases;   Nervous System Diseases;   Genetic Diseases, X-Linked;   Genetic Diseases, Inborn
Interventions: Drug: Ataluren;   Drug: Placebo
7 Completed 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
Intervention: Behavioral: phonetic tests
8 Enrolling by invitation Study of Ataluren for Previously Treated Patients With nmDBMD in the US
Conditions: Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention: Drug: Ataluren
9 Recruiting Study of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Ataluren

Indicates status has not been verified in more than two years