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Dystrophinopathy - 9 Studies Found
Estado | Estudiar |
Completed |
Nombre del estudio: Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids Condición:
Fecha: 2014-04-02 Intervenciones: Other: blood sample dosage of miRNA |
Active, not recruiting |
Nombre del estudio: Clinical Evaluator Outcomes Reliability Study Condición: Dystrophinopathies Fecha: 2014-05-14 |
Active, not recruiting |
Nombre del estudio: Study of Ataluren for Previously Treated Patients With nmDBMD in Europe, Israel, Australia, and Canada Condición:
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Enrolling by invitation |
Nombre del estudio: Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy Condición:
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Completed |
Nombre del estudio: Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy Condición:
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Completed |
Nombre del estudio: Correlation Between Respiratory Impairment and Phonemes Alteration in Dystrophinopathy Patients With Respiratory Failure Condición: During the Medical Appointment, the Speech Therapist Will Perform Phonetic Tests Fecha: 2015-04-03 Intervenciones: Behavioral: phonetic tests |
Enrolling by invitation |
Nombre del estudio: Study of Ataluren for Previously Treated Patients With nmDBMD in the US Condición:
Fecha: 2010-11-22 Intervenciones: 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 |
Nombre del estudio: Study of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy Condición: Duchenne Muscular Dystrophy Fecha: 2016-06-16 Intervenciones: Drug: Ataluren Ataluren Other Names: |