Methylmalonic Acidemia
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: Clinical and Laboratory Study of Methylmalonic Acidemia
This entry reports an ongoing clinical and laboratory study of methylmalonic acidemia, a metabolic disorder, sponsored by the National Human…
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CLINICAL_TRIALS research · 2026-02-25Clinical Trial Update: A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT Gene
This clinical trial update describes a prospective study enrolling pediatric participants up to 16 years with methylmalonic acidemia due to …
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CLINICAL_TRIALS research · 2026-02-05Clinical Trial Update: Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
Long-term follow-up data from patients who received the hLB-001 gene therapy for Methylmalonic Acidemia is being collected, with the study c…
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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 research · 2025-12-23Clinical Trial Update: An 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 [Phase 1, Phase 2]
An extension study to evaluate the long-term safety and clinical activity of mRNA-3705 in participants previously enrolled in other Moderna …
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CLINICAL_TRIALS research · 2025-09-09Clinical Trial Update: A Study to Evaluate the Effects and Safety of Hydroxocobalamin in Participants With Combined Methylmalonic Academia (cblC Type) [Phase 3]
Phase 3 study to evaluate the effects and safety of hydroxocobalamin in participants with combined methylmalonic acidemia (cblC type); the t…
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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 withdrawal · 2024-09-24Clinical Trial Update: A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA [Phase 1, Phase 2]
The clinical trial NCT05778877 evaluating SEL-302 in pediatric patients with methylmalonic acidemia has been withdrawn, with no further enro…
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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…
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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…
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CLINICAL_TRIALS research · 2011-02-08Clinical Trial Update: Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study
This clinical trial update reports on gene identification for CMAMMA and outlines an outcome study. It focuses on malonic aciduria and methy…