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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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Natural History to Assess Disease in Patients With MPS IIIC

NCT07712003

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
Locations: UT Southwestern Children's Medical Center Dallas, Texas, United States

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A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome

NCT07710196

Conditions: Abnormalities, Multiple, Blindness, Congenital Abnormalities, +24 more
Locations: Not specified

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IBS-Oriented CDED Versus Low FODMAP Diet for Irritable Bowel Syndrome

NCT07707635

Conditions: Colonic Diseases, Colonic Diseases, Functional, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +14 more
Locations: Not specified

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Biomarkers for Babies and Young Children With Ataxia Telangiectasia

NCT07709728

Conditions: Ataxia, Ataxia Telangiectasia, Brain Diseases, +19 more
Locations: Not specified

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Luvometinib in Combination With Serplulimab for NF2-Related Tumors

NCT07708285

Conditions: Central Nervous System Neoplasms, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cranial Nerve Diseases, +35 more
Locations: Xuanwu Hospital, Capital Medical University Beijing, Beijing Municipality, China

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

Chinese PLA General Hospital Beijing, Beijing Municipality, China

The First Hospital of Jilin University Changchun, Jilin, China

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Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease

NCT07708350

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations: Boston Children's Hospital Boston, Massachusetts, United States

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Selumetinib for NF2-Related Schwannomatosis

NCT07707947

Conditions: Central Nervous System Neoplasms, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cranial Nerve Diseases, +39 more
Locations: Beijing Tiantan Hospital, Capital Medical University Beijing, Beijing Municipality, China

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Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL)

NCT07654829

Conditions: Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations: KalVista Investigative Site Colorado Springs, Colorado, United States

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Efficacy and Tolerability of a Probiotic Ointment

NCT07701447

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

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Pediatric Von Hippel-Lindau Disease: Natural History, Predictive Factors, and Long-Term Functional Outcomes of Central Nervous System Hemangioblastomas

NCT07705529

Conditions: Abnormalities, Multiple, Angiomatosis, Cardiovascular Diseases, +10 more
Locations: Hôpital Roger Salengro, CHU Lille Lille, France

Hôpital Femme Mère Enfant, HCL Lyon, France

Hôpital Necker Paris, France

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Genetic Diseases, Inborn

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