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Results for “Nonsense Mutations”

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Showing 12 of 0 results

Large-scale testing (Phase 3)Ended earlyNCT02090959
What this trial is testing

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

Who this might be right for
Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, Atrophic+6 more
PTC Therapeutics 219
Early research (Phase 1)Ended earlyNCT02807961
What this trial is testing

Safety, Tolerability, Pharmacokinetics of ELX-02 in Healthy Adult Volunteers

Who this might be right for
Genetic DiseasesNonsense Mutations
Eloxx Pharmaceuticals, Inc. 42
Post-approval studies (Phase 4)UnknownNCT04454151
What this trial is testing

Azithromycin Treatment for Readthrough of APC Gene Stop Codon Mutations in Familial Adenomatous Polyposis

Who this might be right for
Familial Adenomatous Polyposis
Tel-Aviv Sourasky Medical Center 10
Testing effectiveness (Phase 2)Study completedNCT00458341
What this trial is testing

Ataluren in Pediatric Participants With Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 30
Early research (Phase 1)Study completedNCT00005574
What this trial is testing

Gentamicin Treatment of Muscular Dystrophy

Who this might be right for
Becker Muscular DystrophyDuchenne Muscular Dystrophy
National Institute of Neurological Disorders and Stroke (NINDS) 4
Testing effectiveness (Phase 2)UnknownNCT03392909
What this trial is testing

Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Who this might be right for
Recessive Dystrophic Epidermolysis Bullosa
University of Southern California 9
Testing effectiveness (Phase 2)UnknownNCT03492866
What this trial is testing

Efficacy of Topical Gentamycin for Hereditary Hypotrichosis Simplex Caused by Nonsense Mutations in CDSN

Who this might be right for
Hereditary Hypotrichosis Simplex
Tel-Aviv Sourasky Medical Center 8
Large-scale testing (Phase 3)Study completedNCT03179631
What this trial is testing

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, Atrophic+6 more
PTC Therapeutics 360
Large-scale testing (Phase 3)Ended earlyNCT02456103
What this trial is testing

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 246
Testing effectiveness (Phase 2)Study completedNCT05448755
What this trial is testing

ELX-02 in Patients With Alport Syndrome

Who this might be right for
Alport Syndrome
Eloxx Pharmaceuticals, Inc. 3
Testing effectiveness (Phase 2)Ended earlyNCT00759876
What this trial is testing

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 36
Post-approval studies (Phase 4)UnknownNCT02175914
What this trial is testing

Erythromycin Treatment for Readthrough of APC Gene Stop Codon Mutations in Familial Adenomatous Polyposis

Who this might be right for
FAP-Familial Adenomatous Polyposis
michal roll 20