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Clinical Trials for Propionic Acidemia

  • 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
  • Long-term Efficacy of Carglumic Acid in Organic Acidemia.
  • Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
  • Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
  • Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia
  • Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
  • Anaplerotic Therapy in Propionic Acidemia
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