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Clinical Trials for Amino Acid Metabolism, Inborn Errors

• Phase 1/2 Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA
• 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.
• A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
• A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
• Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
• Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency
• EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment
• 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
• Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
• Ataluren for Nonsense Mutation Methylmalonic Acidemia
• Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
• Study of Phosphatidylcholine in a Patient With Methionine Adenosyltransferase Deficiency
• Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders