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Rare Diseases

Explore clinical trials studying Rare Diseases. Study availability and eligibility vary by location and protocol.

473 public trials 86 recruiting studies

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Rare Diseases trial results

Recruiting

The ExTINGUISH Trial of Inebilizumab in NMDAR Encephalitis

NCT04372615

Conditions: Autoimmune Diseases, Autoimmune Diseases of the Nervous System, Autoimmune Encephalitis, +10 more
Locations: University of Alabama at Birmingham Birmingham, Alabama, United States

St. Joseph Hospital and Medical Center Barrow Neurological Institute Phoenix, Arizona, United States

Mayo Clinic in Arizona Scottsdale, Arizona, United States

Children's Hospital of Orange County Orange, California, United States

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Recruiting

RAPA-501 Therapy for ALS

NCT04220190

Conditions: Amyotrophic Lateral Sclerosis, Central Nervous System Diseases, Disease Attributes, +12 more
Locations: Massachusetts General Hospital Boston, Massachusetts, United States

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Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

NCT01793168

Conditions: 1p36 Deletion Syndrome, 3-Methylglutaconic Aciduria, Type V, 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome, +698 more
Locations: Sanford Health Sioux Falls, South Dakota, United States

Online Patient Enrollment System Sydney, Australia

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Recruiting

Brain Aging in Phenylketonuria

NCT06969209

Conditions: Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +14 more
Locations: University Hospital Inselspital, Department of Diabetes, Endocrinology, Nutritional Medicine and Metabolism (UDEM) Bern, Switzerland

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Recruiting

Prospective Observational International Registry of Patients With Newly Diagnosed Peripheral T Cell Lymphoma.

NCT03964480

Conditions: Disease Attributes, Hemic and Lymphatic Diseases, Immune System Diseases, +13 more
Locations: Stanford University Stanford, California, United States

IRCCS Istituto Tumori "Giovanni Paolo II" Bari, Italy

Palermo_La Maddalena Palermo, Italy

Terni-Santa Maria Terni, Italy

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Recruiting

Diagnostic Research in Patients With Rare Diseases -Solving the Unsolved Rare Diseases

NCT04024774

Conditions: Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, +1 more
Locations: CHU de Dijon Dijon, France

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Recruiting

Genetic Causes of Discrepant Clinic in Monogenic Twins

NCT04046796

Conditions: Disease Attributes, Disease Susceptibility, Genetic Predisposition to Disease, +3 more
Locations: University Hospital Tübingen Tübingen, Germany

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Recruiting

The Sarcoma Biology and Outcome Project

NCT04758325

Conditions: Disease Attributes, Malignant Mesenchymoma, Malignant mesenchymal tumor, +11 more
Locations: National Center for Tumour Diseases, University Hospital Heidelberg Heidelberg, Baden-Wurttemberg, Germany

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Recruiting

Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation

NCT06860672

Conditions: Developmental Delay Disorder, Developmental Disabilities, Disease Attributes, +10 more
Locations: Xinhua Hospital affiliated to Shanghai Jiao Tong University School of Medicine Shanghai, Shanghai Municipality, China

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Recruiting

Characteristics of Hypophosphatasia in Adult Patients in Rheumatology and Their Value in Developing an Algorithm to HPP-diagnosis - the COHIR Multi-center Study

NCT06574282

Conditions: Bone Diseases, Bone Diseases, Metabolic, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +11 more
Locations: Clinic of Internal Medicine III, Department of Oncology, Haematology, Rheumatology and Clinical Immunology, University Hospital Bonn Bonn, North Rhine-Westphali, Germany

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