Dystrophinopathy - 9 Studies Found

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

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

: 2010-11-22
: 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 of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy
: Duchenne Muscular Dystrophy
: 2016-06-16
: Drug: Ataluren Ataluren Other Names: