Records containing: Rare Diseases
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CLINICAL_TRIALS study record update · 2026-06-09Evaluation of Safety, Tolerability and Efficacy of Crofelemer Following Multiple Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Participants With Microvillus Inclusion Disease (MVID)
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CLINICAL_TRIALS study record update · 2026-04-27Genomic Sequencing for Evaluation of Uncommon Disease Manifestations Through the Childhood Complex Disease Genomic Section
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CLINICAL_TRIALS study record update · 2026-04-24Genomic Sequencing for Evaluation of Uncommon Disease Manifestations Through the Childhood Complex Disease Genomic Section
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CLINICAL_TRIALS study record update · 2026-04-24Longitudinal Studies of Patients and Families With Familial Platelet Disorders With Associated Myeloid Malignancy (FPDMM) Caused by RUNX1 Germline Variants or FPDMM-Like Conditions
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CLINICAL_TRIALS study record update · 2026-04-14Evaluation of Socio-professional Inclusion for Young Adults Aged 15-25 Living With a Rare Genetic Disability
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CLINICAL_TRIALS study record update · 2026-04-14A Multicenter Non Interventional Single Arm Retrospective-prospective Observational Study in Therapeutic Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis (gMG) in Real Clinical Practice in Russia
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CLINICAL_TRIALS study record update · 2026-04-13Using Social Robots to Improve the Relationship and Alleviate Anxiety and Stress in Children With Rare Diseases and Their Parents: A Feasibility Study
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CLINICAL_TRIALS study record update · 2026-04-01BEhavioral and Adherence Model for Improving Quality, Health Outcomes and Cost-Effectiveness of healthcaRe
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CLINICAL_TRIALS study record update · 2026-03-27Family Adaptation and Relationship Dynamics in Pediatric Rare Diseases.
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CLINICAL_TRIALS study record update · 2026-02-19A Multicenter Non-interventional Single-arm Retrospective-prospective Observational Study in Therapeutic Approaches on AQP4-IgG Positive Neuromyelitis Optica Spectrum Disorder (NMOSD) in Real Clinical Practice in Russia
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CLINICAL_TRIALS study record update · 2026-02-12Efficacy of Rheopheresis as Adjuvant Treatment of Calciphylaxis in Hemodialysis Patients : a Prospective Randomized Controled Single-blind Trial
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CLINICAL_TRIALS study record update · 2026-02-10Implementation of Long-read Sequencing for Epimutation Detection in the Diagnosis of Rare Diseases.
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CLINICAL_TRIALS study record update · 2026-02-06Preferences and Representations Concerning High-throughput Sequencing Technologies in Medical Genetics. The Case of Development Anomalies.
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CLINICAL_TRIALS study record update · 2026-01-27MAGNET: Making Genomics Accessible For Newborns In Texas
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CLINICAL_TRIALS study record update · 2026-01-14Institutional Registries of Rare Diseases at Hospital Italiano de Buenos Aires (HIBA)
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CLINICAL_TRIALS study record update · 2025-12-16UNIQUE: Umbrella N-of-1 Tumor Trials - Umbrella Protocol for Oncology Single Patient Protocols
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CLINICAL_TRIALS study record update · 2025-12-15Rare and Undiagnosed Disease Research Biorepository
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CLINICAL_TRIALS study record update · 2025-12-05Identification of Cellular Biomarkers of Rare Eye Diseases in Adults
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CLINICAL_TRIALS study record update · 2025-11-28Swiss Rare Disease Registry
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CLINICAL_TRIALS study record update · 2025-11-19Evaluation of the French Version of the PEERS Social Skills Training Program for Adolescents With Rare Neurodevelopmental Disorders / PEERS-MRND
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CLINICAL_TRIALS study record update · 2025-10-03Auto-immune Thrombotic Thrombocytopenic Purpura : Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center, TWI-LIGHT
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CLINICAL_TRIALS study record update · 2025-09-12Oral Health Related Quality of Life of Patients With Rare Diseases Followed at Necker Hospital: a Qualitative Approach
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CLINICAL_TRIALS study record update · 2025-09-10FIBROBLASTS AND THORACIC AORTIC ANEURYSMS: IN VITRO CHARACTERIZATION IN PATIENTS WITH MARFAN SYNDROME AND GENETIC AORTIC DISEASES
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CLINICAL_TRIALS study record update · 2025-09-10Aortic Stiffness in Patients With Genetic Aortopathies: Intervention Analysis
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CLINICAL_TRIALS study record update · 2025-09-10Analysis of Muscle Morphological and Electromechanical Properties in Patients With MFS and EDS: a Low-risk Intervention Study