Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

Propionic acidemia

A Retrospective Study to Characterize Participants With Propionic Acidemia Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM) A Long-Term Extension Study to Evaluate the Safety and Clinical Activity of mRNA-3927 A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Open Label Study of mRNA-3927 in Patients With Propionic Acidemia Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia Review of Charts From Amish/Mennonite Variant PA Patients “The MaP Study”: Mapping the Patient Journey in MMA and PA Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia Anaplerotic Therapy in Propionic Acidemia Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia