Inborn Errors of Metabolism
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol
This update outlines a pregnancy registry protocol to study maternal outcomes in pregnancies affected by inborn errors of metabolism, includ…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: Clinical and Laboratory Study of Methylmalonic Acidemia
This entry reports an ongoing clinical and laboratory study of methylmalonic acidemia, a metabolic disorder, sponsored by the National Human…
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CLINICAL_TRIALS research · 2026-03-10Clinical Trial Update: Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital
This clinical trial is evaluating therapeutic adherence among patients with hereditary/metabolic disorders at Necker Hospital; the study is …
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CLINICAL_TRIALS research · 2026-02-20Clinical Trial Update: Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC [EARLY_Phase 1]
An early-phase expanded-access protocol evaluating CD3+/CD19+ depleted peripheral blood stem cells for leukemia and related immunodeficiency…
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CLINICAL_TRIALS research · 2026-01-15Clinical Trial Update: Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders
This completed clinical trial update from Duke University describes identifying and characterizing novel non-coding genetic variants that co…
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CLINICAL_TRIALS research · 2026-01-14Clinical Trial Update: Institutional Registry of Rare Diseases
An institutional registry for rare diseases sponsored by Hospital Italiano de Buenos Aires is currently recruiting. The registry includes am…
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CLINICAL_TRIALS research · 2025-11-20Clinical Trial Update: Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
Clinical trial update describing expanded access to T-cell-depleted haplo-identical stem cells for patients undergoing haplo-identical or un…
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CLINICAL_TRIALS research · 2024-08-06Clinical Trial Update: Targeted Interventions for Successful Transition and Transfer of Adolescents With Inborn Errors of Metabolism to Adult Services [NA]
This clinical trial update describes a study of targeted interventions to help adolescents with inborn errors of metabolism transition to ad…
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CLINICAL_TRIALS research · 2020-07-08Clinical Trial Update: North Carolina Newborn Exome Sequencing for Universal Screening [NA]
A completed clinical trial from UNC Chapel Hill evaluating newborn exome sequencing for universal screening, with results posted in 2020.