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Refsum Disease, Infantile - 8 Studies Found

Status Study
Terminated Study Name: Study of Bile Acids in Patients With Peroxisomal Disorders
Condition:
  • Infantile Refsum's Disease
  • Zellweger Syndrome
  • Drug: chenodeoxycholic acid
  • Drug: cholic a
Completed Study Name: Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
Condition:
  • Infantile Refsum's Disease
  • Zellweger Syndrome
  • Mucopolysaccharidosis Disorders
  • Hurler Syndrome
  • Hunter Syn
    Date: 2014-06-20
    Interventions:
    • Biological: Stem Cell Transplantation

Completed Study Name: Betaine and Peroxisome Biogenesis Disorders
Condition: Peroxisome Biogenesis Disorders
Date: 2013-03-29
Interventions: Drug: Betaine Betaine will be given orally (mixed with foo
Terminated Study Name: Study of Bile Acids in Patients With Peroxisomal Disorders
Condition:
  • Infantile Refsum's Disease
  • Zellweger Syndrome
  • Drug: chenodeoxycholic acid
  • Drug: cholic a
Completed Study Name: Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
Condition:
  • Infantile Refsum's Disease
  • Zellweger Syndrome
  • Mucopolysaccharidosis Disorders
  • Hurler Syndrome
  • Hunter Syn
    Date: 2014-06-20
    Interventions:
    • Biological: Stem Cell Transplantation

Completed Study Name: Betaine and Peroxisome Biogenesis Disorders
Condition: Peroxisome Biogenesis Disorders
Date: 2013-03-29
Interventions: Drug: Betaine Betaine will be given orally (mixed with foo