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Genetic Diseases, X-Linked

Clinical trial condition

Genetic Diseases, X-Linked

Explore clinical trials studying Genetic Diseases, X-Linked. Study availability and eligibility vary by location and protocol.

1,244 public trials 227 recruiting studies

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Genetic Diseases, X-Linked trial results

Completed

A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome

NCT03098797

Conditions: Abnormalities, Multiple, Barth Syndrome, Cardiovascular Abnormalities, +12 more
Locations: McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine Baltimore, Maryland, United States

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Completed

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

NCT04279314

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations: University of Alabama at Birmingham Birmingham, Alabama, United States

Translational Genomics Research Institute (TGen) Phoenix, Arizona, United States

University of California, San Diego La Jolla, California, United States

UC Davis MIND Institute Sacramento, California, United States

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Completed

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

NCT04181723

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations: University of Alabama at Birmingham Birmingham, Alabama, United States

Translational Gemomics Research Institute (TGen) Phoenix, Arizona, United States

University of California, San Diego La Jolla, California, United States

UC Davis MIND Institute Sacramento, California, United States

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Completed

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

NCT04804566

Conditions: Angiokeratoma, Brain Diseases, Brain Diseases, Metabolic, +37 more
Locations: Engage Health Eagan, Minnesota, United States

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Completed

Assessment of Nutritional and Growth Status in Rett Syndrome Patients

NCT06335368

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +8 more
Locations: Not specified

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Completed

Effectiveness of Aquatic Therapy in Children With Duchenne and Becker Muscular Dystrophy

NCT06186310

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne or Becker Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations: Gaziosmanpasa Research and Education Hospital Istanbul, Gaziosmanpaşa, Turkey (Türkiye)

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Completed

Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI

NCT01569568

Conditions: Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +15 more
Locations: Not specified

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Completed

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

NCT02667483

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations: Kobe University Hospital Hyōgo, Kobe-shi, Japan

National Center of Neurology and Psychiatry Tokyo, Kodaira-shi, Japan

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Completed

Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE (HOPE Fabry)

NCT05671770

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Cleveland Clinic AbuDhabi Abu Dhabi, United Arab Emirates

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Completed

Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and B

NCT03276130

Conditions: Anxiety Disorders, Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, +16 more
Locations: Swedish Orphan Biovitrum Research Site Copenhagen, Denmark

Swedish Orphan Biovitrum Research Site Helsinki, Finland

Swedish Orphan Biovitrum Research Site Gothenburg, Sweden

Swedish Orphan Biovitrum Research Site Malmö, Sweden

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Genetic Diseases, X-Linked

Genetic Diseases, X-Linked trial results

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