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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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Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)

NCT07684105

Conditions: Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +18 more
Locations: CHU de Nice Nice, France

APHP Necker Paris, France

APHP St Louis Paris, France

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Not Yet Recruiting

Low Dose Bolus Ketamine For Use In Sickle Cell Pain Crisis

NCT07682662

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations: University of Mississippi Medical Center, Pediatric Emergency Department Jackson, Mississippi, United States

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A Phase 2 Study of the Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy

NCT07680322

Conditions: Brain Diseases, Central Nervous System Diseases, Congenital Abnormalities, +21 more
Locations: Not specified

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European Cystinosis Cohort 2

NCT07680751

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, Genetic Diseases, Inborn, +4 more
Locations: AP-HP_ Hôpital Charles Foix Ivry-sur-Seine, Île-de-France Region, France

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Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)

NCT07682129

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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Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease

NCT07641140

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: The First Affiliated Hospital, Zhejiang University School of Medicine Hangzhou, Zhejiang, China

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The Safety and Efficacy of Upadacitinib in Refractory Autoimmune Related Cholangitis and Atopic Dermatitis With Moderate to Severe Itching

NCT07678645

Conditions: Atopic Dermatitis (AD), Bile Duct Diseases, Biliary Tract Diseases, +27 more
Locations: RenJi Hospital Shanghai, Shanghai Municipality, China

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Assessing Molecular Mechanisms and Effects of Music Therapy in Youth With Sickle Cell Disease Using Single-cell RNA-sequencing

NCT07562451

Conditions: Agnosia, Anemia, Anemia, Hemolytic, +14 more
Locations: Children's Healthcare of Atlanta Atlanta, Georgia, United States

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

NCT07290530

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

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Concurrent Training vs Soleus Push-Ups on Neurogenesis-Related Biomarkers in Diabetic Neuropathy Patients

NCT07675720

Conditions: Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diabetes Complications, +17 more
Locations: Pakistan Railways General Hospital and Mediplex Healthcare Center Rawalpindi, Pakistan

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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