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Inborn Amino Acid Metabolism Disorder - 25 Studies Found

Status Study
Suspended Study Name: Ataluren for Nonsense Mutation Methylmalonic Acidemia
Condition: Amino Acid Metabolism, Inborn Errors
Date: 2010-06-07
Interventions: Drug: Ataluren (PTC124) Ataluren (PTC124) will be provided as a vanilla-flavored powder to be mixed with
Completed Study Name: Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders
Condition: Amino Acid Metabolism, Inborn Errors
Date: 2000-02-24
Interventions:
  • Drug: Sodium Benzoate
  • Drug: Sodium Phenyla
Completed Study Name: Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
Condition: Amino Acid Metabolism, Inborn Errors
Date: 1999-10-18
Interventions:
  • Behavioral: Protein and calorie controlled diet
Completed Study Name: Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
Condition:
  • Argininosuccinic Aciduria
  • Amino Acid Metabolism, Inborn Errors<
    Date: 2006-06-26
    Interventions:
    • Drug: Sodium Phenylbutyrate No
Recruiting Study Name: Longitudinal Study of Urea Cycle Disorders
Condition:
  • Brain Diseases, Metabolic, Inborn
  • Amino Acid Metabolism, Inborn Errors
    Date: 2005-10-10
Recruiting Study Name: A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
Condition: Aromatic Amino Acid Decarboxylase Deficiency
Date: 2016-10-03
Interventions: Drug: AAV2-hAADC Dosage form: Aqueous solutionDose(s): 2.371x1011 vg/case Dosing schedule: Intracerebral
Recruiting Study Name: A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
Condition: AADC Deficiency
Date: 2016-07-20
Interventions: Drug: AAV2-hAADC Subjects will be enrolled sequentially in
Not yet recruiting Study Name: Review of French Cases of Glutathione Synthetase Deficiency
Condition: The Glutathione Synthetase Deficiency
Date: 2016-06-17
Completed Study Name: Newborn Screening for Aromatic L-amino Acid Decarboxylase Deficiency
Condition: Aromatic L-amino Acid Decarboxylase Deficiency
Date: 2015-03-21
Completed Study Name: PET Imaging of GABA Receptors in Succinic Semialdehyde Dehydrogenase Deficiency
Condition:
  • Succinic Semialdehyde Dehydrogenase Deficiency
  • SSADH Deficiency

Date: 2005-10-29
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