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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.

12,578 public trials 2,158 recruiting studies

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Genetic Diseases, Inborn trial results

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Phase 4 Master Protocol for Patients Prescribed Prademagene Zamikeracel for the Treatment of Wounds

NCT07717736

Conditions: Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations: Not specified

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An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

NCT07750990

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations: Not specified

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Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal Dystrophy

NCT07750678

Conditions: Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +20 more
Locations: University of California, Los Angeles (UCLA) Los Angeles, California, United States

Fudan University Eye & ENT Hospital Shanghai, Shanghai Municipality, China

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Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.

NCT07465718

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: University of Colorado Anschutz School of Medicine Denver, Colorado, United States

Yale University School of Medicine New Haven, Connecticut, United States

University of Michigan Medical Centre Ann Arbor, Michigan, United States

Beijing YouAn Hospital, Capital Medical University Beijing, Beijing Municipality, China

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Optimizing Parameters of Transcranial Temporal Interference Stimulation for Spinocerebellar Ataxia Type 3

NCT07743866

Conditions: Ataxia, Brain Diseases, Central Nervous System Diseases, +14 more
Locations: Department of Rehabilitation Medicine, The First Affiliated Hospital of Fujian Medical University Fuzhou, Fujian, China

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Combination of Avutometinib and Defactinib for Treatment of Relapsed, Refractory, Metastatic, or Unresectable Malignant Peripheral Nerve Sheath Tumor

NCT07743554

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fibrosarcoma, Genetic Diseases, Inborn, +26 more
Locations: Not specified

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A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS)

NCT07746089

Conditions: CMS, Congenital Myasthenic Syndrome, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +5 more
Locations: Not specified

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Epidermal Growth Factor Receptor Inhibition for Keratinopathies

NCT06545695

Conditions: Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +16 more
Locations: Department of Dermatology, Northwestern University Feinberg School of Medicine Chicago, Illinois, United States

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Study Aiming to Test Whether Non-invasive Liquid Biopsies Can Safely Reduce Invasive Surveillance Methods in Lynch Syndrome

NCT07360834

Conditions: Colonic Diseases, Colorectal Neoplasms, Colorectal Neoplasms, Hereditary Nonpolyposis, +16 more
Locations: Klinicki Bolnicki Centar Sestre Milosrdnice Ustanova Zagreb, Croatia

Masaryk Memorial Cancer Institute Brno, Czechia

Tampereen Korkeakoulusäätiö SR Helsinki, Finland

GGC Network Paris, France

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Effectiveness of Upadacitinib and Biologics for the Achievement of Minimal Disease Activity in Adults With Moderate-to-Severe Atopic Dermatitis (RAMpUp)

NCT07741838

Conditions: Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations: Not specified

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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