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Genetic Diseases, Inborn

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Genetic Diseases, Inborn

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

12,537 public trials 2,139 recruiting studies

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Genetic Diseases, Inborn trial results

Recruiting

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44

NCT07037862

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy (DMD), Genetic Diseases, Inborn, +8 more
Locations: University Hospital Gent Ghent, Belgium

UZ Leuven Leuven, Belgium

Centre Hospitalier Régional de la Citadelle Liège, Belgium

IRCCS Ospedale San Raffaele Milan, Italy

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Recruiting

A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia

NCT05669950

Conditions: Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +16 more
Locations: University Hospital-University of Michigan Ann Arbor, Michigan, United States

Rigshospitalet Copenhagen, Denmark

Chu Angers Angers, France

CHU de Lille Lille, France

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Recruiting

Longitudinal Biomarkers With Selected Outcome Measures In CMT

NCT06794489

Conditions: CMT (Charcot Marie Tooth Disease), CMT - Charcot-Marie-Tooth Disease, CMT 1A, +13 more
Locations: University Medical Centre Göttingen, Lower Saxony, Germany

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Recruiting

CAT-VHL Exploring the Role of Carbonic Anhydrase IX as Diagnostic and Theranostic Target in Von-Hippel Lindau Disease

NCT07171905

Conditions: Abnormalities, Multiple, Angiomatosis, Cardiovascular Diseases, +9 more
Locations: IRCCS Ospedale San Raffaele Milan, Italia, Italy

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Recruiting

Observational Database on Deep Brain Stimulation in Tourette Syndrome

NCT04449068

Conditions: Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +10 more
Locations: CIC Neurosciences, GH Pitié-Salpêtrière Paris, France

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Recruiting

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

NCT05996003

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DMD, Duchenne Muscular Dystrophy, +10 more
Locations: Children's Hospital Colorado Aurora, Colorado, United States

Rare Disease Research, LLC - FL Kissimmee, Florida, United States

Rare Disease Research Atlanta, Georgia, United States

Ann and Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States

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Metabolic Impacts of Ren-Nu: A Dietary Program for Polycystic Kidney Disease

NCT07454174

Conditions: Abnormalities, Multiple, Autosomal Dominant Polycystic Kidney Disease (ADPKD), Ciliopathies, +12 more
Locations: Cleveland Clinic Cleveland, Ohio, United States

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Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study

NCT07301216

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: Yale School of Medicine New Haven, Connecticut, United States

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Recruiting

Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)

NCT07247058

Conditions: ACTH Syndrome, Ectopic, Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, +23 more
Locations: Johns Hopkins University School of Medicine Baltimore, Maryland, United States

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Natural History Study of Patients With Hypophosphatasia (HPP)

NCT02237625

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Hypophosphatasia, +4 more
Locations: Duke University Medical Center Durham, North Carolina, United States

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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