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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.

12,559 public trials 2,153 recruiting studies

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Genetic Diseases, Inborn trial results

Recruiting

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

NCT06421831

Conditions: Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +9 more
Locations: Beijing Tiantan Hospital, Capital Medical University Beijing, Beijing Municipality, China

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Recruiting

Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease

NCT07049588

Conditions: Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth Disease Type 1A, Congenital Abnormalities, +10 more
Locations: Assistance Publique - Hôpitaux de Marseille Marseille, France

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Recruiting

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

NCT06391736

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations: Chinese PLA General Hospital Beijing, China

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Recruiting

Trial of JMKX003142 in Participants With Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)

NCT06800651

Conditions: ADPKD (Autosomal Dominant Polycystic Kidney Disease), Abnormalities, Multiple, Ciliopathies, +12 more
Locations: Peking University First Hospital Beijing, Beijing Municipality, China

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Recruiting

Progression Assessment of PXE-associated Alterations

NCT07048106

Conditions: Angioid Streaks, Cardiovascular Diseases, Congenital Abnormalities, +17 more
Locations: Department of Ophthalmology, University Hospital Bonn, Germany Bonn, North Rhine-Westphalia, Germany

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Recruiting

A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)

NCT06563414

Conditions: Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +20 more
Locations: Krystal Biotech Pittsburgh, Pennsylvania, United States

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Recruiting

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

NCT06224413

Conditions: Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +22 more
Locations: Boston Children's Hospital Boston, Massachusetts, United States

Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States

UT Southwestern Medical Center Dallas, Texas, United States

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Recruiting

Oral Deucrictibant for Prophylactic and Acute Treatment in Hereditary Angioedema Patients

NCT07046806

Conditions: Angioedema, Angioedemas, Hereditary, Bradykinin-mediated Angioedema, +16 more
Locations: Institute For Asthma & Allergy Wheaton, Maryland, United States

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Recruiting

Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

NCT07046338

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Shenzhen Geno-Immune Medical Institute Shenzhen, Guangdong, China

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Recruiting

Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration

NCT07040137

Conditions: Brain Diseases, Central Nervous System Diseases, Cerebellar Diseases, +8 more
Locations: Research Site Multiple Locations, Japan

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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