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Results for “Nonsense Mutations”

Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT03648827
What this trial is testing

Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 20
Testing effectiveness (Phase 2)Study completedNCT02819557
What this trial is testing

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 14
Early research (Phase 1)Study completedNCT03292302
What this trial is testing

Phase 1 Study of ELX-02 in Healthy Adults

Who this might be right for
Genetic DiseaseNonsense Mutation
Eloxx Pharmaceuticals, Inc. 18
Post-approval studies (Phase 4)UnknownNCT02354560
What this trial is testing

Erythromycin Treatment for Readthrough of APC Gene Stop Codon Mutation in Familial Adenomatous Polyposis-minors' Adjusted Version

Who this might be right for
FAP-Familial Adenomatous Polyposis
michal roll 15
Testing effectiveness (Phase 2)Ended earlyNCT01009294
What this trial is testing

Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

Who this might be right for
Duchenne Muscular DystrophyBecker Muscular Dystrophy
PTC Therapeutics 6
Testing effectiveness (Phase 2)Study completedNCT03796637
What this trial is testing

Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 6
Testing effectiveness (Phase 2)Study completedNCT03012191
What this trial is testing

Gentamicin for RDEB

Who this might be right for
Recessive Dystrophic Epidermolysis Bullosa
University of Southern California 6
Testing effectiveness (Phase 2)Study completedNCT00237380
What this trial is testing

Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 24
Testing effectiveness (Phase 2)Study completedNCT00351078
What this trial is testing

PTC124 for the Treatment of Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 19
Testing effectiveness (Phase 2)Study completedNCT04644627
What this trial is testing

Topical Gentamicin Nonsense Suppression Therapy of EB

Who this might be right for
Epidermolysis Bullosa
Oslo University Hospital 4
Testing effectiveness (Phase 2)Study completedNCT00264888
What this trial is testing

Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 38
Large-scale testing (Phase 3)Study completedNCT00803205
What this trial is testing

Study of Ataluren (PTC124™) in Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 238
Post-approval studies (Phase 4)Study completedNCT03256799
What this trial is testing

Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations

Who this might be right for
Cystic Fibrosis
University of Alabama at Birmingham 1
Large-scale testing (Phase 3)Study completedNCT01247207
What this trial is testing

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 270
Not applicableStudy completedNCT04174469
What this trial is testing

Ivermectin Neurotoxicity and ABCB1 Gene Mutations

Who this might be right for
DNA Sequencing
University Hospital, Montpellier 3
Testing effectiveness (Phase 2)Study completedNCT00234663
What this trial is testing

PTC124 for Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 24
Testing effectiveness (Phase 2)Ended earlyNCT00947193
What this trial is testing

Study of Ataluren (PTC124) in Hemophilia A and B

Who this might be right for
Hemophilia AHemophilia B
PTC Therapeutics 13
Testing effectiveness (Phase 2)WithdrawnNCT04117880
What this trial is testing

Open Label Extension Study in Participants With Nonsense Mutation Aniridia

Who this might be right for
Aniridia
PTC Therapeutics
Testing effectiveness (Phase 2)Looking for participantsNCT03526159
What this trial is testing

Gentamicin for Junctional Epidermolysis Bullosa

Who this might be right for
Junctional Epidermolysis Bullosa
University of Southern California 6
Testing effectiveness (Phase 2)Looking for participantsNCT07523581
What this trial is testing

EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety

Who this might be right for
Alport Syndrome, X-LinkedAlport Syndrome, Autosomal Recessive
Eloxx Pharmaceuticals, Inc. 24
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