Propionic Acidemia
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
Ongoing clinical study updates describe natural history, physiology, microbiome, and biochemistry investigations in propionic acidemia; recr…
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CLINICAL_TRIALS research · 2026-03-23Clinical Trial Update: A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study [Phase 1, Phase 2]
A clinical trial update describing a long-term safety and clinical activity study of mRNA-3927 in participants previously enrolled in the mR…
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CLINICAL_TRIALS research · 2026-02-05Clinical Trial Update: A Retrospective Study to Characterize Participants With Propionic Acidemia
This study is a retrospective analysis to characterize participants with Propionic Acidemia.
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CLINICAL_TRIALS trial_result · 2026-01-29Clinical Trial Update: Review of Charts From Amish/Mennonite Variant PA Patients
Completed clinical trial update reviewing charts from Amish/Mennonite variant Propionic Acidemia patients.
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CLINICAL_TRIALS research · 2026-01-22Clinical Trial Update: Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia [Phase 1, Phase 2]
Open-label Phase 1/2 trial evaluating mRNA-3927 for propionic acidemia is currently recruiting.
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EMA approval · 2025-12-09EMA Authorised: Carbaglu (carglumic acid) — Tour Hekla
The European Medicines Agency has authorised Carbaglu (carglumic acid) for propionic acidemia; the marketing authorization holder is Tour He…
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CLINICAL_TRIALS research · 2025-04-15Clinical Trial Update: An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
An observational study update evaluating Carbaglu (carglumic acid) for the treatment of hyperammonemia in adults and children with methylmal…
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CLINICAL_TRIALS trial_result · 2025-03-20Clinical Trial Update: Long-term Efficacy of Carglumic Acid in Organic Acidemia. [NA]
A completed study assessing the long-term efficacy of carglumic acid in organic acidemias. The conditions include Propionic Acidemia and Met…
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CLINICAL_TRIALS research · 2024-10-30Clinical Trial Update: MyRareDiet A Novel Diet Tracking Tool [NA]
This clinical trial update outlines MyRareDiet, a novel diet-tracking tool being studied for urea cycle disorders and related organic acidem…
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CLINICAL_TRIALS trial_result · 2021-02-15Clinical Trial Update: Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia [Phase 2]
Phase 2 trial updates short-term outcomes of N-Carbamylglutamate for acute hyperammonemia in several metabolic disorders; results posted aft…
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CLINICAL_TRIALS trial_result · 2019-04-25Clinical Trial Update: Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia [Phase 3]
Phase 3 trial update for Carglumic Acid in Propionic Acidemia and Methylmalonic Acidemia has completed.
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CLINICAL_TRIALS trial_result · 2017-04-26Clinical Trial Update: Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia [Phase 2]
Phase 2 trial examining long-term outcomes of N-Carbamylglutamate in Propionic Acidemia and Methylmalonic Acidemia; the study was terminated…
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CLINICAL_TRIALS trial_result · 2015-01-21Clinical Trial Update: Anaplerotic Therapy in Propionic Acidemia [Phase 1]
A Phase 1 study evaluating anaplerotic therapy for propionic acidemia has completed.
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CLINICAL_TRIALS withdrawal · 2014-05-29Clinical Trial Update: Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate [Phase 2]
Phase 2 trial of N-Carbamylglutamate to increase ureagenesis in urea cycle disorders and other inborn errors of metabolism has been withdraw…