Records containing: Methylmalonic Acidemia (MMA)
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CLINICAL_TRIALS study record update · 2026-02-25A Prospective, Longitudinal, Observational, Multi-centre Study of Pediatric Participants up to 16 Years of Age With Methylmalonyl-CoA Mutase Deficiency Caused by Mutations in the MMUT Gene That Results in a Diagnosis of Isolated Methylmalonic Acidemia (MMA).
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CLINICAL_TRIALS study record update · 2025-09-09A Single-arm Phase III Clinical Study to Evaluate the Efficacy and Safety of Hydroxocobalamin Chloride Injection in Participants With Methylmalonic Acidemia (MMA) With Elevated Homocysteine (Cobalamin C Deficiency)
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CLINICAL_TRIALS study record update · 2024-09-24Phase 1/2 Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 (MMA-101 Following Administration of SEL-110) in Pediatric Subjects With Mut Subtype Isolated Methylmalonic Acidemia (MMA)
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CLINICAL_TRIALS study record update · 2020-11-13A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency