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Rare Diseases

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

467 public trials 83 recruiting studies

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Rare Diseases trial results

Not Yet Recruiting

Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)

NCT07560956

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
Locations: Sahlgrenska University Hospital Gothenburg, Sweden

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Not Yet Recruiting

Implementing Dynamic Consent for Rare Disease Patients

NCT06656247

Conditions: Behavior, Disease Attributes, Health Behavior, +7 more
Locations: Not specified

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Not Yet Recruiting

A Longitudinal Study on Family Adaptation and Relationship Dynamics in Pediatric Rare Diseases

NCT07497581

Conditions: Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, +1 more
Locations: University Children's Hospital Zurich Zurich, Switzerland

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Not Yet Recruiting

Implementation of Long-read Sequencing for the Diagnosis of Rare Diseases.

NCT07400913

Conditions: Albinism, Amino Acid Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +22 more
Locations: CHU Bordeaux - Hôpital Pellegrin Bordeaux, France

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Not Yet Recruiting

TEGSEDI Pregnancy Surveillance Program

NCT04270058

Conditions: Amyloidosis, Hereditary, Transthyretin-Related, Disease Attributes, Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy, +4 more
Locations: Not specified

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Not Yet Recruiting

Channelling Algorithm Proficiency To Understand, Recognise and Elucidate Disease

NCT07130071

Conditions: Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, +2 more
Locations: Not specified

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Not Yet Recruiting

Initiative for Clinical Long-read Sequencing

NCT06060184

Conditions: Disease Attributes, Disease Susceptibility, Genetic Predisposition, +4 more
Locations: University Hospital Tübingen Tübingen, Germany

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Completed

HIBA-Institutional Registry of Amyloidosis

NCT01347047

Conditions: Amyloidosis, Disease Attributes, Metabolic Diseases, +5 more
Locations: Hospital Italiano de Buenos Aires Buenos Aires, Argentina

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Completed

Interleukin-1 Receptor Antagonist (IL-1RA) (ANAKINRA) IN SEVERE SYSTEMIC-ONSET JUVENILE IDIOPATHIC ARTHRITIS

NCT00339157

Conditions: Arthritis, Arthritis, Juvenile, Autoimmune Diseases, +11 more
Locations: Bordeaux CHU Bordeaux, France

Hopital Lyon Edouard Herriot Lyon, France

Nancy Hopital d'Enfants Nancy, France

Pediatric Immuno-Hematology and Rheumatology Unit, Necker-Enfants Malades Hospital, 149 rue de Sevres Paris, France

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Completed

Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

NCT02116764

Conditions: Chronic Disease, Chronic Granulomatous Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +13 more
Locations: National Institutes of Health Clinical Center Bethesda, Maryland, United States

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Rare Diseases

Rare Diseases trial results

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