26 studies found for:    ataluren
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Identifiers
Dates
Rank Status Study
1 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
2 Not yet recruiting Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
Condition: Epilepsy
Interventions: Drug: ataluren;   Drug: Placebo
3 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)
4 Completed 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
5 Terminated 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)
6 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
7 Completed Study of Ataluren (PTC124™) in Cystic Fibrosis
Condition: Cystic Fibrosis
Interventions: Drug: Ataluren (PTC124);   Drug: Placebo
8 Terminated Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Ataluren
9 Enrolling by invitation Extension Study of Ataluren for Nonsense Mutation Cystic Fibrosis
Condition: Cystic Fibrosis
Intervention: Drug: Ataluren
10 Completed Extension Study of Ataluren (PTC124) in Cystic Fibrosis
Condition: Cystic Fibrosis
Intervention: Drug: Ataluren (PTC124)
11 Suspended Study of Ataluren (PTC124®) in Hemophilia A and B
Conditions: Hemophilia A;   Hemophilia B
Intervention: Drug: Ataluren (PTC124)
12 Recruiting Study of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Ataluren
13 Suspended Ataluren for Nonsense Mutation Methylmalonic Acidemia
Condition: Amino Acid Metabolism, Inborn Errors
Intervention: Drug: Ataluren (PTC124)
14 Active, not recruiting Study of Ataluren (PTC124) in Cystic Fibrosis
Condition: Cystic Fibrosis
Intervention: Drug: Ataluren (PTC124)
15 Recruiting Study of Ataluren in Patients With Aniridia
Condition: Aniridia
Interventions: Drug: Ataluren;   Drug: Placebo
16 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
17 Completed Phase 2b Study of PTC124 in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Conditions: Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention: Drug: PTC124
18 Not yet recruiting Effects of Rifampin on the Pharmacokinetics of Ataluren
Condition: Healthy
Interventions: Drug: Ataluren;   Drug: Rifampin
19 Active, not recruiting Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
Condition: Cystic Fibrosis
Interventions: Drug: Ataluren (PTC124®);   Drug: Placebo
20 Completed PTC124 for the Treatment of Cystic Fibrosis
Condition: Cystic Fibrosis
Intervention: Drug: PTC124

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Indicates status has not been verified in more than two years