Inborn Errors of Metabolism
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-04-06Clinical Trial Update: Inherited Diseases, Caregiving, and Social Networks
NHGRI-sponsored trials investigating undiagnosed diseases, inherited metabolic and neurodegenerative disorders, and Alzheimer's-related deme…
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CLINICAL_TRIALS trial_result · 2025-12-08Clinical Trial Update: Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases [Phase 2]
A Phase 2 trial comparing treosulfan-based conditioning to busulfan-based conditioning in pediatric patients with non-malignant diseases has…
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CLINICAL_TRIALS trial_result · 2025-05-13Clinical Trial Update: North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2 [NA]
ClinicalTrials.gov update indicates the UNC Chapel Hill genomic evaluation study using next-generation exome sequencing has completed and po…
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CLINICAL_TRIALS trial_result · 2025-03-30Clinical Trial Update: ORCHARD- Optimising Home Assessment of Rural Patients [NA]
ORCHARD, a trial on Optimising Home Assessment of Rural Patients, has completed; the update provides no trial results in this notice.
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CLINICAL_TRIALS trial_result · 2024-12-04Clinical Trial Update: Orphan Drugs for Inherited Metabolic Diseases
A trial update from the University of Lausanne indicates completion of studies on orphan drugs for inherited metabolic diseases. The snippet…
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CLINICAL_TRIALS trial_result · 2024-12-04Clinical Trial Update: Cardiac Manifestation and Inherited Metabolic Diseases
A completed clinical trial evaluating cardiac manifestations in patients with inborn errors of metabolism.
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CLINICAL_TRIALS trial_result · 2020-02-17Clinical Trial Update: N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia [Phase 2, Phase 3]
Phase 2/3 trial update for N-Carbamylglutamate (Carbaglu) in the treatment of hyperammonemia due to inborn errors of metabolism.
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CLINICAL_TRIALS trial_result · 2018-08-21Clinical Trial Update: CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant [Phase 2]
Phase 2 clinical trial update on CD34+ selection for partially matched family or matched unrelated adult donor transplants in leukemia and l…
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CLINICAL_TRIALS trial_result · 2017-06-29Clinical Trial Update: Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells [Phase 1]
A Phase 1 trial of unrelated umbilical cord blood transplantation augmented with ALDHbr cord blood cells has completed, with the update desc…
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CLINICAL_TRIALS trial_result · 2017-03-09Clinical Trial Update: Protein Sorbent Properties of Montmorillonite in Vitro and in Vivo Models [NA]
A completed study evaluating montmorillonite's protein-sorbent properties in vitro and in vivo models relevant to urea cycle disorders and o…
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CLINICAL_TRIALS trial_result · 2016-03-15Clinical Trial Update: Anti-oxLDL IgM Antibodies as a Novel Therapy for Metabolic Lipid Diseases [EARLY_Phase 1]
An early-phase, first-in-humans trial is testing anti-oxLDL IgM antibodies as a potential treatment for lipid metabolism disorders arising f…
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CLINICAL_TRIALS trial_result · 2014-12-09Clinical Trial Update: Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders [NA]
A completed clinical trial evaluating total body irradiation and fludarabine followed by allogeneic transplantation with cyclosporine and my…
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CLINICAL_TRIALS trial_result · 2014-08-20Clinical Trial Update: The Early History of Universal Screening for Metabolic Disorders
This completed clinical trial update from the University of Miami provides historical context on the early universal screening programs for …
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CLINICAL_TRIALS trial_result · 2014-08-13Clinical Trial Update: Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders [Phase 1]
Clinical trial update on reduced intensity conditioning for umbilical cord blood transplantation in pediatric patients with non-malignant di…
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CLINICAL_TRIALS trial_result · 2014-07-08Clinical Trial Update: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases [Phase 3]
Phase 3 trial of ALD-101 as adjuvant therapy with unrelated umbilical cord blood transfusion in inherited metabolic diseases was terminated.