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17 studies found for:    ataluren

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1 Terminated Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Conditions: Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention: Drug: Ataluren (PTC124)
2 Suspended Study of Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
Conditions: Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention: Drug: Ataluren (PTC124)
3 Active, not recruiting Extension Study of Ataluren (PTC124) in Cystic Fibrosis
Condition: Cystic Fibrosis
Intervention: Drug: Ataluren (PTC124)
4 Suspended Study of Ataluren (PTC124®) in Hemophilia A and B
Conditions: Hemophilia A;   Hemophilia B
Intervention: Drug: Ataluren (PTC124)
5 Completed Study of Ataluren (PTC124™) in Cystic Fibrosis
Condition: Cystic Fibrosis
Interventions: Drug: Ataluren (PTC124);   Drug: Placebo
6 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 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
8 Suspended Ataluren for Nonsense Mutation Methylmalonic Acidemia
Condition: Amino Acid Metabolism, Inborn Errors
Intervention: Drug: Ataluren (PTC124)
9 Terminated Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Ataluren
10 Recruiting Study of Ataluren for Previously Treated Patients With nmDBMD in the US
Conditions: Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention: Drug: Ataluren
11 Completed Phase 2b Study of PTC124 in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Conditions: Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention: Drug: PTC124
12 Completed PTC124 for the Treatment of Cystic Fibrosis
Condition: Cystic Fibrosis
Intervention: Drug: PTC124
13 Completed PTC124 for Cystic Fibrosis
Condition: Cystic Fibrosis
Intervention: Drug: PTC124
14 Completed PTC124 for Pediatric Patients With Cystic Fibrosis
Condition: Cystic Fibrosis
Intervention: Drug: PTC124
15 Completed Safety and Efficacy of PTC124 for Cystic Fibrosis
Condition: Cystic Fibrosis
Intervention: Drug: PTC124
16 Completed Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: PTC124
17 Recruiting The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor
Condition: Duchenne Muscular Dystrophy
Intervention:
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† Indicates status has not been verified in more than two years