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genetic diseases, inborn

Showing 1 - 25 of 32
STUDY
CONDITIONS
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LAST UPDATED

Healthy Volunteer, Muscular Dystrophies, Muscular Disorders, Atrophic Trial in Madison (EDG-5506 Tablet, Radiolabeled EDG-5506

Recruiting
  • Healthy Volunteer
  • +7 more
  • EDG-5506 Tablet
  • +2 more
  • Madison, Wisconsin
    Labcorp Clinical Research Unit, Inc.
Feb 7, 2023

Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn Trial in Nashville (CTX001)

Recruiting
  • Beta-Thalassemia
  • +4 more
  • CTX001
  • Nashville, Tennessee
  • +5 more
Feb 2, 2023

Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic Trial in Little Rock, Sacramento, Atlanta

Recruiting
  • Muscular Dystrophies
  • +7 more
  • CAP-1002
  • Placebo
  • Little Rock, Arkansas
  • +9 more
Jan 9, 2023

Prader-Willi Syndrome, Aggression, Self-Injurious Behavior Trial in Brooklyn (Guanfacine Extended Release, Placebo)

Recruiting
  • Prader-Willi Syndrome
  • +20 more
  • Guanfacine Extended Release
  • Placebo
  • Brooklyn, New York
    Maimonides Medical Center
Dec 20, 2022

Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn Trial in Worldwide (CTX001)

Active, not recruiting
  • Beta-Thalassemia
  • +4 more
  • CTX001
  • Stanford, California
  • +11 more
Nov 30, 2022

A Long-term Follow-up Study in Subjects Who Received CTX001

Enrolling by invitation
  • Beta-Thalassemia
  • +6 more
  • CTX001
  • New York, New York
  • +12 more
Nov 18, 2022

DM1, Muscular Dystrophies, Myotonic Dystrophy Trial in United States (Placebo, AOC 1001)

Enrolling by invitation
  • DM1
  • +12 more
  • Placebo
  • AOC 1001
  • Los Angeles, California
  • +7 more
Jul 27, 2022

Beta-Thalassemia, Thalassemia, Hematologic Diseases Trial (CTX001)

Not yet recruiting
  • Beta-Thalassemia
  • +6 more
  • CTX001
  • (no location specified)
Jul 26, 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

Evaluating Prenatal Exome Sequencing Study

Enrolling by invitation
  • Congenital Anomalies
  • +2 more
    • Leiden, Zuid-Holland, Netherlands
      Leiden University Medical Centre
    Mar 21, 2022

    Pilot Study of Cardiac MR in Patients With Muscular Dystrophy

    Recruiting
    • Muscular Dystrophies
    • +3 more
      • Washington, District of Columbia
        Children's National Health System
      Mar 1, 2022

      Data of Current FVIII Prophylaxis Replacement Therapy in Adult

      Active, not recruiting
      • Blood Coagulation Disorder
      • +8 more
      • Standard of Care FVIII Replacement therapy
      • San Francisco, California
      • +10 more
      Feb 18, 2022

      Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited Trial in United States

      Recruiting
      • Adeno-Associated Virus (AAV)
      • +16 more
      • SPK-8016
      • Los Angeles, California
      • +13 more
      Feb 3, 2022

      Dermatitis, Atopic, Dermatitis, Eczema Trial in Hamilton (Benralizumab, Placebo Control)

      Completed
      • Dermatitis, Atopic
      • +9 more
      • Hamilton, Ontario, Canada
        McMaster Cardio-Respiratory Research Lab
      Oct 14, 2021

      Human Growth Hormone Treatment in Small for Gestational Age

      Not yet recruiting
      • Small for Gestational Age Infant
      • +2 more
      • Blood collection for genetic analysis
      • (no location specified)
      Sep 27, 2021

      Dermatitis, Dermatitis, Atopic, Eczema Trial in Worldwide (PF-04965842 100 mg, PF-04965842 200 mg, Placebo)

      Completed
      Has Results
      • Dermatitis
      • +9 more
      • PF-04965842 100 mg
      • +2 more
      • Birmingham, Alabama
      • +234 more
      Sep 17, 2021

      Utilizing Free DNA in Embryo Culture for PGT

      Completed
      • Chromosome Abnormalities
      • +2 more
      • NGS reading for Media Free DNA
      • (no location specified)
      Aug 13, 2021

      Children With Metachromatic Leukodystrophy

      Terminated
      • Lipid Metabolism Disorders
      • +13 more
        • Torrance, California
        • +13 more
        Mar 16, 2021

        Whole Genome Sequencing in the Neonatal Intensive Care Unit

        Completed
        • Genetic Diseases, Inborn
        • Sequence
        • Hershey, Pennsylvania
          Penn State Health Milton S. Hershey Medical Center
        Jan 29, 2021

        Dermatitis, Dermatitis, Atopic, Eczema Trial in Worldwide (PF-04965842 100 mg, PF-04965842 200 mg, Dupilumab)

        Completed
        Has Results
        • Dermatitis
        • +9 more
        • PF-04965842 100 mg
        • +4 more
        • Birmingham, Alabama
        • +215 more
        Dec 21, 2020

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

        Terminated
        Has Results
        • Muscular Dystrophy, Duchenne
        • +8 more
        • Los Angeles, California
        • +57 more
        Aug 10, 2020

        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

        Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic Trial in United States (CAP-1002, Placebo)

        Completed
        • Muscular Dystrophies
        • +7 more
        • CAP-1002
        • Placebo
        • Sacramento, California
        • +6 more
        Jun 3, 2020

        Neurodevelopment Dysplasia of Fetal Brain Caused by ASNS Gene

        Unknown status
        • Asparagine Synthetase Deficiency
        • Genetic Diseases, Inborn
          • Chang Chun, Jilin, China
            Bo Chen
          Jul 3, 2018

          Symptomatic Domains Most Relevant to Charcot Marie Tooth

          Completed
          • Charcot Marie Tooth Disease (CMT)
          • +6 more
            • Tampa, Florida
              RDCRN Data Management and Coordinating Center , Epidemiology Cen
            Jun 9, 2017