Methylmalonic Acidemia
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CLINICAL_TRIALS trial_result · 2026-01-20Clinical Trial Update: A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia [Phase 1, Phase 2]
This is a clinical trial update for the investigational mRNA therapy mRNA-3705 to assess safety, pharmacokinetics, and pharmacodynamics in p…
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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 · 2024-02-23Clinical Trial Update: Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia [Phase 1, Phase 2]
A Phase 1/2 clinical trial of gene therapy hLB-001 in pediatric patients with methylmalonic acidemia has terminated with results posted.
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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 · 2020-07-07Clinical Trial Update: Ataluren for Nonsense Mutation Methylmalonic Acidemia [Phase 2]
Phase 2 clinical trial of ataluren for nonsense mutation-induced methylmalonic acidemia has been terminated; results posted.
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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…