Records containing: Inborn Errors of Metabolism
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CLINICAL_TRIALS study record update · 2026-04-20Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol
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CLINICAL_TRIALS study record update · 2026-04-15Phase 1/2 Study: CD45RA Depleted Peripheral Stem Cell Addback to Prevent Viral and Fungal Infections Following Alternative Donor TCRab/CD19 Depleted Hematopoietic Stem Cell Transplant
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CLINICAL_TRIALS study record update · 2026-04-08Clinical and Basic Investigations of Methylmalonic Acidemia (MMA) and Related Disorders
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CLINICAL_TRIALS study record update · 2026-02-20Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated Donor or Related Donor Peripheral Stem Cells
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CLINICAL_TRIALS study record update · 2026-01-15Identification and Characterization of Novel Coding, Splicing and Non-Coding Variants That Contribute to Genetic Disorders
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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 · 2025-12-08Clinical Phase II Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) in Paediatric Patients With Non-malignant Diseases
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CLINICAL_TRIALS study record update · 2025-05-13North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2
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CLINICAL_TRIALS study record update · 2025-03-30Optimising Remote Consulting and Home Assessment of Medically Vulnerable Rural Patients During Unscheduled and Planned Primary Care- Assessing Feasibility of the ORCHARD Intervention
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CLINICAL_TRIALS study record update · 2024-12-04The Challenge of Treating Orphan Diseases With Novel Therapies: Experience From One Adult Metabolic Clinic in Switzerland
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CLINICAL_TRIALS study record update · 2024-10-23Growth and Development of Children With Inborn Errors of Metabolism in Assiut Governorate
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CLINICAL_TRIALS study record update · 2024-08-06Fit for Transfer: Targeted Interventions for Successful Transition and Transfer of Adolescents With Inborn Errors of Metabolism to Adult Metabolic Services
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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 · 2024-03-01Enterobacteria Strain-level Characterization in Children With Inborn Errors of Metabolism (IEM)
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CLINICAL_TRIALS study record update · 2024-02-16A Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence.
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CLINICAL_TRIALS study record update · 2024-02-16A Study to Evaluate the Acceptability of a New Phenylalanine Free Infant Formula for Use in the Dietary Management of Phenylketonuria in Infants From Birth to 2 Year of Age With Regard to Product Tolerance and Adherence.
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CLINICAL_TRIALS study record update · 2023-10-11The Safety, Tolerability, Pharmacodynamics, and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase (EE-TP) in Patients With MNGIE
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CLINICAL_TRIALS study record update · 2023-02-10Induced Pluripotent Stem Cells for the Development of Novel Drug Therapies for Inborn Errors of Metabolism, an International, Multicenter Study
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CLINICAL_TRIALS study record update · 2022-07-05Utilizing Augmented Artificial Intelligence for Aminoacidopathies: Experience of Collaborative Laboratory Integrated Reporting From Pakistan
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CLINICAL_TRIALS study record update · 2022-03-24Biomarkers for Inborn Errors of Metabolism: An International, Multicenter, Observational, Longitudinal Protocol
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CLINICAL_TRIALS study record update · 2021-12-09A Collection of Case Studies in Infants With Urea Cycle Disorders (UCD) to Evaluate Infant Growth and the Safety of a New Medical Food for UCD
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CLINICAL_TRIALS study record update · 2021-07-20Clinical Characteristics of Adult Patients With Inborn Errors of Metabolism in French-speaking Switzerland
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CLINICAL_TRIALS study record update · 2021-01-14Evaluating the Clinical Utility of Face-Recognition Technology in Syndrome Diagnosis
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CLINICAL_TRIALS study record update · 2020-11-16Protecting Brains and Saving Futures - the PBSF Protocol: a Prospective Multicenter and Observational Study on the Use of Telemedicine for Neurocritical Care in High-risk Newborns in Brazil.
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CLINICAL_TRIALS study record update · 2020-11-04Identification and Validation of Relevant Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism