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 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 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.