Propionic Acidemia

• CLINICAL_TRIALS research · 2026-04-20
  Clinical 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…

• CLINICAL_TRIALS research · 2026-03-23
  Clinical 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…

• CLINICAL_TRIALS research · 2026-02-05
  Clinical Trial Update: A Retrospective Study to Characterize Participants With Propionic Acidemia

  This study is a retrospective analysis to characterize participants with Propionic Acidemia.

• CLINICAL_TRIALS research · 2026-01-22
  Clinical 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.

• CLINICAL_TRIALS research · 2025-04-15
  Clinical 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…

• Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
• CLINICAL_TRIALS research · 2024-10-30
  Clinical 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…