Records containing: Rare Diseases
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2024-11-22SLC13A5 Deficiency: a Prospective Natural History Study - Remote Only (International)
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CLINICAL_TRIALS study record update · 2024-11-19Clinical, Instrumental and Laboratory Data Collection of Subjects with Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases
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CLINICAL_TRIALS study record update · 2024-11-14FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
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CLINICAL_TRIALS study record update · 2024-10-01Rare Tumours in Children and Adolescents (STEP 2.0) - Register for the Documentation of Rare Tumours in Children and Adolescents
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CLINICAL_TRIALS study record update · 2024-08-30Epilepsy Learning Healthcare System (ELHS) for Quality Care & Outcome Improvement
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CLINICAL_TRIALS study record update · 2024-05-14Validation of Human Drugs Target of Repurposed Drugs and Novel Therapies
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CLINICAL_TRIALS study record update · 2024-05-08Evaluating the Therapeutic Efficacy of Filgrastim in Severe Bullous Drug Eruptions (Lyell and Stevens-Johnson Syndromes)
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CLINICAL_TRIALS study record update · 2024-04-12Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases
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CLINICAL_TRIALS study record update · 2024-01-12Psychological Help in Patients With Marfan Syndrome: the Role of Quality of Life, Coping Mechanisms, Psychosocial Aspects and Self-esteem Levels
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CLINICAL_TRIALS study record update · 2023-12-13Duale Lotsenstruktur Zur Abklärung Unklarer Diagnosen in Zentren für Seltene Erkrankungen
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CLINICAL_TRIALS study record update · 2023-11-29Identification of the Genetic Causes of Rare Diseases With Negative Exome Findings
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CLINICAL_TRIALS study record update · 2023-11-29Genome-based Management of Patients in Precision Medicine (Ge-Med) Towards a Genomic Health Program
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CLINICAL_TRIALS study record update · 2023-11-24A Randomized, Double-blinded, Placebo-controlled, Single Center, Phase I Study to Evaluate Pharmacokinetics, Pharmacodynamics and Safety of Emapalumab After a Single Intravenous Dose in Japanese Healthy Volunteers.
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CLINICAL_TRIALS study record update · 2023-11-13Phase I Trial of Pbi-shRNA™ EWS/FLI1 Type 1 Lipoplex (LPX) in Subjects With Advanced Ewing's Sarcoma
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CLINICAL_TRIALS study record update · 2023-08-04Vocal Pattern Assessment as a New Key to Identifying Rare Syndromes
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CLINICAL_TRIALS study record update · 2023-08-03A Phase I-II, FIH Study of A166 in Locally Advanced/Metastatic Solid Tumors Expressing Human Epidermal Growth Factor Receptor 2 (HER2) or Are HER2 Amplified That Did Not Respond or Stopped Responding to Approved Therapies
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CLINICAL_TRIALS study record update · 2023-04-26Clinical Safety and Efficacy of Pharmacogenetics in Veteran Care
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CLINICAL_TRIALS study record update · 2023-04-26A Multi-Center Phase III, Randomized, Open-Label Trial of Vigil (Bi-shRNAfurin and GMCSF Augmented Autologous Tumor Cell Immunotherapy) in Combination With Irinotecan and Temozolomide as a Second-Line Regimen for Ewing's Sarcoma
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CLINICAL_TRIALS study record update · 2023-04-18Open-label Pilot Study to Assess the Efficacy and Safety of Cannabidiol Oral Solution as an Adjunctive Treatment for Children and Young Adults With Rare Disease-associated Severe Epilepsy
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CLINICAL_TRIALS study record update · 2023-04-04National Cheng Kung University Hospital
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CLINICAL_TRIALS study record update · 2023-01-26Efficacy of Intact Cord Resuscitation Compared to Immediate Cord Clamping on Cardiorespiratory Adaptation at Birth in Infants With Isolated Congenital Diaphragmatic Hernia (CDH)
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CLINICAL_TRIALS study record update · 2023-01-11Gewinnung, Kultur Und Studien Von primären Humanen Zelltypen, Geweben Und Blut für Die Gezielte Behandlung Von Seltenen Hauterkrankungen
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CLINICAL_TRIALS study record update · 2022-11-10A Lifecourse Map of Common Diseases and Rare Diseases in China
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CLINICAL_TRIALS study record update · 2022-10-18Whole Genome Trio Sequencing as a Standard Routine Test in Patients With Rare Diseases - "GENOME FIRST APPROACH"
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CLINICAL_TRIALS study record update · 2022-09-16Children Affected by Rare Disease and Their Families Network