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Genetic Diseases, X-Linked

Showing 1 - 25 of >10,000
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X-Linked Dystonia Parkinsonism Trial in Manila (transcranial magnetic resonance-guided focused ultrasound pallidothalamic

Available
  • X-Linked Dystonia Parkinsonism
  • transcranial magnetic resonance-guided focused ultrasound pallidothalamic tractotomy
  • Manila, Philippines
    Philippine General Hospital
Aug 21, 2022

Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and

Recruiting
  • Granuloma
  • +4 more
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Jan 25, 2023

    Exosome for Early Diagnosis of Bipolar Affective Disorder

    Not yet recruiting
    • Bipolar Affective Disorder
    • Major Depressive Disorder
      • Tianjin, China
        Tianjin Anding Hospital
      Jun 13, 2023

      Fragile X Syndrome, Fragile X Mental Retardation Syndrome, Mental Retardation, X Linked Trial in Sacramento (Placebo Medication,

      Active, not recruiting
      • Fragile X Syndrome
      • +9 more
      • Sacramento, California
        UC Davis MIND Institute
      Feb 22, 2022

      Study of Smith-Lemli-Opitz Syndrome

      Completed
      • Smith-Lemli-Opitz Syndrome
      • Cholesterol Suspension
      • +2 more
      • Bethesda, Maryland
        National Institutes of Health Clinical Center
      Jan 25, 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

      Biochemical and Phenotypical Aspects of Smith-Lemli-Opitz

      Recruiting
      • Smith Lemli Opitz Syndrome
      • +3 more
        • Bethesda, Maryland
          National Institutes of Health Clinical Center
        Aug 17, 2022

        Fragile X Syndrome, Fragile X Mental Retardation Syndrome, Mental Retardation, X-Linked Trial in Edmonton, Montreal (Placebo

        Recruiting
        • Fragile X Syndrome
        • +9 more
        • Edmonton, Alberta, Canada
        • +1 more
        Sep 16, 2021

        Rett Syndrome, Rett Syndrome, Atypical, Genetic Disease Trial in United States

        Recruiting
        • Rett Syndrome
        • +9 more
          • Birmingham, Alabama
          • +13 more
          Jul 12, 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

          ESRD, Fabry Disease, Rare Diseases Trial in Boston

          Terminated
          • ESRD
          • +5 more
            • Boston, Massachusetts
              Massachusetts General Hospital
            Feb 22, 2021

            Smith-Lemli-Opitz Syndrome, Cone-Rod Dystrophy, Hearing Loss Trial in Aurora (Antioxidants, Cholesterol)

            Recruiting
            • Smith-Lemli-Opitz Syndrome
            • +2 more
            • Aurora, Colorado
              Children's Hospital Colorado
            Jan 4, 2022

            Bipolar Disorder, Bipolar Depression, Bipolar I Disorder Trial in Stockholm (Aerobic Physical Exercise, Relaxing Physical

            Not yet recruiting
            • Bipolar Disorder
            • +5 more
            • Aerobic Physical Exercise
            • +2 more
            • Stockholm, Sweden
              Region Stockholm, Psykiatri Sydväst (Psychiatric Clinic Psychiat
            Apr 26, 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

            Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP) Trial in Worldwide (Dersimelagon, Placebo)

            Not yet recruiting
            • Erythropoietic Protoporphyria (EPP)
            • X-Linked Protoporphyria (XLP)
            • Huntington Beach, California
            • +25 more
            Nov 17, 2023

            Clinical and Genetic Studies of X-Linked Juvenile Retinoschisis

            Recruiting
            • Retinoschisis
              • Bethesda, Maryland
                National Institutes of Health Clinical Center
              Jan 25, 2023

              X-Linked Dystonia Parkinsonism Trial in Roxas City (MINGO)

              Completed
              Has Results
              • X-Linked Dystonia Parkinsonism
              • MINGO
              • Roxas City, Capiz, Philippines
                Health Centrum
              Apr 20, 2020

              Severe Combined Immunodeficiency Disease, X-linked Trial in San Francisco, Memphis, Seattle (genetic, drug, device)

              Recruiting
              • Severe Combined Immunodeficiency Disease, X-linked
              • CL20-i4-EF1α-hγc-OPT
              • +2 more
              • San Francisco, California
              • +2 more
              Jan 9, 2023

              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

              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

              X-Linked Retinitis Pigmentosa Trial in Worldwide (Genetic: AAV5-RPGR 4e11, Genetic: AAV5-RPGR 2e11)

              Recruiting
              • X-Linked Retinitis Pigmentosa
              • Genetic: AAV5-RPGR 4e11
              • Genetic: AAV5-RPGR 2e11
              • Los Angeles, California
              • +9 more
              Aug 16, 2022

              Pediatric Patients With Metabolic or Other Genetic Disorders

              Recruiting
              • Genetic Disorder
              • +4 more
                • Bethesda, Maryland
                  National Institutes of Health Clinical Center
                Jan 28, 2023

                X-linked Hypophosphatemia, Hypophosphatemic Rickets, Hypophosphatemic Rickets, X-Linked Dominant Trial in Boston (Calcitriol)

                Recruiting
                • X-linked Hypophosphatemia
                • +2 more
                • Boston, Massachusetts
                  Massachusetts General Hospital
                Mar 16, 2022

                X-linked Severe Combined Immunodeficiency (XSCID) Trial run by the National Institute of Allergy and Infectious Diseases (NIAID)

                Suspended
                • X-linked Severe Combined Immunodeficiency (XSCID)
                • Bethesda, Maryland
                  National Institutes of Health Clinical Center, 9000 Rockville Pi
                Aug 24, 2022

                Effort Test asTherapeutic Monitoring Tool in Acute

                Completed
                • Rhabdomyolysis Linked to a Hereditary Disease of Metabolism
                • Effort test
                • Functional tests on fibroblasts
                • Paris, France
                  Hôpital Necker-Enfants Malades
                Oct 10, 2022