Records containing: Propionic Acidemia
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CLINICAL_TRIALS study record update · 2026-04-20The Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
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CLINICAL_TRIALS study record update · 2026-03-23A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
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CLINICAL_TRIALS study record update · 2026-02-05A Retrospective Cohort Study to Characterize Propionic Acidemia Patients
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CLINICAL_TRIALS study record update · 2026-01-29Chart Review of Patients Who Have the Amish/Mennonite Variant of Propionic Acidemia
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CLINICAL_TRIALS study record update · 2026-01-22A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants With Propionic Acidemia
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CLINICAL_TRIALS study record update · 2025-08-12Universal Genomic Newborn Screening in the Wallonia-Brussels Federation: Baby Detect
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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 · 2025-04-04Early Check: A Collaborative Innovation to Facilitate Pre-Symptomatic Clinical Trials in Newborns
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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 · 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 · 2015-01-21Safety & Efficacy of Investigational Products: Ornithine Alpha-ketoglutarate, Glutamine, or Disodium Citrate on Hyperammonemia in Propionic Acidemia.
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CLINICAL_TRIALS study record update · 2014-05-29Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate