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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.

449 public trials 107 recruiting studies

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Genetic Diseases, Inborn trial results

Completed

Clinical Research Core Dental Screening Protocol

NCT00090818

Conditions: Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, +14 more
Locations: National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland, United States

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Completed

Identification of Genes Associated With Lung Disease in Patients With Rheumatoid Arthritis

NCT00001885

Conditions: Arthritis, Arthritis, Rheumatoid, Autoimmune Diseases, +22 more
Locations: National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland, United States

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Completed

Genomic Sequencing in Acutely Ill Neonates

NCT02225522

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease, Diseases/Diagnoses, +4 more
Locations: Children's Mercy Hospital Kansas City, Missouri, United States

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Completed

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

NCT02053064

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +13 more
Locations: Hôpitaux Universitaires Paris Sud (Bicêtre) Le Kremlin-Bicêtre, France

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Completed

Cord Blood Transplantation in Severe Aplastic Anemia

NCT01343953

Conditions: Absence of an HLA Identical Donor, Acquired Aplastic Anemia, Anemia, +13 more
Locations: saint Louis Paris, France

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Completed

An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients

NCT02429947

Conditions: Charcot Marie Tooth Disease (CMT), Charcot-Marie-Tooth Disease, Congenital Abnormalities, +13 more
Locations: RDCRN Data Management and Coordinating Center , Epidemiology Center; University of South Florida Tampa, Florida, United States

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Completed

Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis

NCT00004533

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +9 more
Locations: Not specified

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Completed

A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin

NCT01435629

Conditions: Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +6 more
Locations: Novo Nordisk Investigational Site Tokyo, Japan

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Completed

Study Utilizing Rilonacept in Gout Exacerbations

NCT00855920

Conditions: Acute Gout Flare, Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations: Birmingham, Alabama, United States

Huntsville, Alabama, United States

Mesa, Arizona, United States

Phoenix, Arizona, United States

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Completed

Review of Safety Using Rilonacept in Preventing Gout Exacerbations (RE-SURGE)

NCT00856206

Conditions: Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
Locations: Alabaster, Alabama, United States

Mesa, Arizona, United States

Burbank, California, United States

Long Beach, California, United States

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