Records containing: Methylmalonic Acidemia
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 · 2026-04-08Clinical and Basic Investigations of Methylmalonic Acidemia (MMA) and Related Disorders
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CLINICAL_TRIALS study record update · 2026-02-05A Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic Acidemia Characterized by MMUT Mutations Who Received hLB-001
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CLINICAL_TRIALS study record update · 2026-01-20A Global Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
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CLINICAL_TRIALS study record update · 2025-12-23A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
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CLINICAL_TRIALS study record update · 2025-04-15A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations
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CLINICAL_TRIALS study record update · 2024-10-30MyRareDiet™: A Diet Tracking, Monitoring and Optimization mHealth Tool for Patients With Inborn Errors of Metabolism
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CLINICAL_TRIALS study record update · 2024-02-23A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations
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CLINICAL_TRIALS study record update · 2024-02-05Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
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CLINICAL_TRIALS study record update · 2024-01-05A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Followed by a Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study and an Open-label, Long-term Extension Study
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CLINICAL_TRIALS study record update · 2023-12-27JUMP, Journey to Understand Methylmalonic Acidemia and Propionic Acidemia - a Natural History Study
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CLINICAL_TRIALS study record update · 2023-12-21A First-in-human, Randomized, Placebo-controlled, Single and Multiple Ascending Dose Escalation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BBP-671 in Healthy Subjects and In Patients With Propionic Acidemia or Methylmalonic Acidemia
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CLINICAL_TRIALS study record update · 2021-08-02A Longitudinal, Exploratory, Natural History Study to Further Characterize and Describe the Signs and Symptoms of Patients With Organic Acidemias
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CLINICAL_TRIALS study record update · 2021-02-15Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
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CLINICAL_TRIALS study record update · 2019-04-25Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carbaglu® in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)
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CLINICAL_TRIALS study record update · 2017-04-26Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia
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CLINICAL_TRIALS study record update · 2014-05-29Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
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CLINICAL_TRIALS study record update · 2011-02-08ClinicalTrials.gov study NCT01289158