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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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CS-206 in Patients With Sickle Cell Disease

NCT07000318

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations: Children's Hospital of Fudan University Shanghai, Shanghai Municipality, China

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Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)

NCT07326592

Conditions: Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations: Not specified

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Retrospective Identification of Scintigraphic Cardiac Amyloidosis (RISCA)

NCT06921408

Conditions: Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +15 more
Locations: Not specified

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Amyloid Lowering for Alzheimer's in Down's With Donanemab Investigation

NCT06911944

Conditions: Abnormalities, Multiple, Alzheimer Disease, Amyloid Beta Protein, +21 more
Locations: Not specified

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Gene Therapy Trial for CLN6 Batten Disease

NCT07582484

Conditions: Batten Disease, Batten's Disease, CLN6, +14 more
Locations: University of California, San Diego - Rady Children's La Jolla, California, United States

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Minocycline In Neurocognitive Outcomes - Sickle Cell Disease

NCT05605366

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +21 more
Locations: Not specified

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Ultrasound Measurement of Thyroid Volume in Term Newborns

NCT07579988

Conditions: Bone Diseases, Bone Diseases, Developmental, Bone Diseases, Endocrine, +11 more
Locations: IRCCS Ospedale San Raffaele Milan, Italy

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Early Screening for Cardiac Amyloid Using a History of Bilateral Carpal Tunnel Syndrome

NCT07580872

Conditions: Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +17 more
Locations: Not specified

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A Study of SNH-119014 in Adult Participants With Non-transfusion-dependent Thalassemia (NTDT)

NCT07579949

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +6 more
Locations: Foshan First People's Hospital Foshan, Guangdong, China

Nanfang Hospital, Southern Medical University Guangzhou, Guangdong, China

Maoming People's Hospital Maoming, Guangdong, China

Liuzhou People's Hospital Liuzhou, Guangxi, China

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Screening, Characterization, and Longitudinal Follow-up of Patients With Cardiac Amyloidosis

NCT07577466

Conditions: Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +25 more
Locations: University Hospital Duesseldorf Düsseldorf, North Rhine-Westphalia, Germany

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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