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Nonsense mutation

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Nonsene Mutation Duchenne Muscular Dystrophy Trial in Atlanta (Ataluren)

Recruiting
  • Nonsene Mutation Duchenne Muscular Dystrophy
  • Atlanta, Georgia
    Rare Disease Research, LLC
Jan 27, 2023

Duchenne Muscular Dystrophy Trial in Los Angeles (Ataluren)

Completed
Has Results
  • Duchenne Muscular Dystrophy
  • Los Angeles, California
    University of California, Los Angeles (UCLA)
Mar 9, 2022

Alport Syndrome Trial in Clayton, Parkville, London (ELX-02)

Recruiting
  • Alport Syndrome
  • Clayton, Victoria, Australia
  • +3 more
Dec 19, 2022

Epilepsy Trial in New York (ataluren, Placebo)

Completed
  • Epilepsy
  • New York, New York
    New York University School of Medicine
Sep 7, 2021

Duchenne Muscular Dystrophy Trial in Canada, United States (Ataluren)

Enrolling by invitation
  • Duchenne Muscular Dystrophy
  • Phoenix, Arizona
  • +32 more
Jan 31, 2023

Anatomic Stage IV Breast Cancer AJCC v8, Metastatic HER2-Negative Breast Carcinoma, Metastatic Hormone Receptor-Positive Breast

Not yet recruiting
  • Anatomic Stage IV Breast Cancer AJCC v8
  • +2 more
  • (no location specified)
Nov 8, 2022

Duchenne Muscular Dystrophy Trial in United States (Ataluren)

Completed
Has Results
  • Duchenne Muscular Dystrophy
  • Phoenix, Arizona
  • +8 more
Mar 10, 2022

Genetic Disease, Nonsense Mutation Trial in Antwerp (ELX-02, Placebo)

Completed
Has Results
  • Genetic Disease
  • Nonsense Mutation
  • Antwerp, Belgium
    SGS Life Sciences, Clinical Pharmacology Unit
Feb 23, 2021

Duchenne Muscular Dystrophy, Becker Muscular Dystrophy Trial in United Kingdom, United States (Ataluren, Chronic Corticosteroid

Terminated
Has Results
  • Duchenne Muscular Dystrophy
  • Becker Muscular Dystrophy
  • Davis, California
  • +5 more
Jul 27, 2020

Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy Trial in Worldwide (Ataluren)

Completed
Has Results
  • Duchenne Muscular Dystrophy
  • +2 more
  • Parkville, Melbourne, Australia
  • +20 more
Nov 3, 2020

Duchenne Muscular Dystrophy Trial in Cincinnati, Philadelphia, Salt Lake City (Ataluren)

Terminated
Has Results
  • Duchenne Muscular Dystrophy
  • Cincinnati, Ohio
  • +2 more
Oct 2, 2020

Amino Acid Metabolism, Inborn Errors Trial in Worldwide (Ataluren)

Terminated
Has Results
  • Amino Acid Metabolism, Inborn Errors
  • Antwerp, Belgium
  • +8 more
Jun 22, 2020

Cystic Fibrosis Trial in Jerusalem (Ataluren)

Completed
  • Cystic Fibrosis
  • Jerusalem, Israel
    Hadassah University Hospital - Mount Scopus
Jun 10, 2020

Duchenne Muscular Dystrophy Trial in United States (Ataluren)

Completed
Has Results
  • Duchenne Muscular Dystrophy
  • Gulf Breeze, Florida
  • +5 more
Aug 12, 2020

Genetic Disease, Nonsense Mutation, Cystinosis Trial in Montréal (ELX-02)

Terminated
  • Genetic Disease
  • +2 more
  • MontrĂ©al, Quebec, Canada
    McGill University Health Center
May 22, 2020

Cystic Fibrosis Trial in Birmingham (Ataluren)

Completed
Has Results
  • Cystic Fibrosis
  • Birmingham, Alabama
    University of Alabama at Birmingham
Mar 25, 2020

Cystic Fibrosis Trial in Brussels, Leuven, Paris (Ataluren)

Completed
Has Results
  • Cystic Fibrosis
  • Brussels, Belgium
  • +2 more
Feb 21, 2020

Aniridia Trial (Ataluren)

Withdrawn
  • Aniridia
  • (no location specified)
Oct 3, 2019

Muscular Dystrophy, Duchenne Trial in Worldwide

Active, not recruiting
  • Muscular Dystrophy, Duchenne
    • Linz, Austria
    • +70 more
    Aug 16, 2022

    Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic Trial in Worldwide (Ataluren, PLACEBO)

    Active, not recruiting
    • Muscular Dystrophy, Duchenne
    • +8 more
    • Phoenix, Arizona
    • +71 more
    Mar 30, 2022

    Recessive Dystrophic Epidermolysis Bullosa Trial (Gentamicin, Placebo)

    Completed
    Has Results
    • Recessive Dystrophic Epidermolysis Bullosa
    • (no location specified)
    Oct 15, 2019

    Hemophilia A, Hemophilia B Trial in Worldwide (Ataluren)

    Terminated
    Has Results
    • Hemophilia A
    • Hemophilia B
    • Peoria, Illinois
    • +12 more
    May 28, 2020

    Cystic Fibrosis Trial in Worldwide (Ataluren)

    Completed
    Has Results
    • Cystic Fibrosis
    • Birmingham, Alabama
    • +30 more
    Oct 16, 2020

    Cystic Fibrosis Trial in Australia, Germany, Israel (ELX-02, Ivacaftor)

    Recruiting
    • Cystic Fibrosis
    • Camperdown, New South Whales, Australia
    • +8 more
    Feb 15, 2022

    Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic Trial in Worldwide (Ataluren, Placebo)

    Completed
    Has Results
    • Muscular Dystrophy, Duchenne
    • +8 more
    • Los Angeles, California
    • +54 more
    Jul 17, 2020