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Barth Syndrome Summary

Description: Rare congenital X-linked disorder of lipid metabolism. Barth syndrome is transmitted in an X-linked recessive pattern. The syndrome is characterized by muscular weakness, growth retardation, DILATED CARDIOMYOPATHY, variable NEUTROPENIA, 3-methylglutaconic aciduria (type II) and decreases in mitochondrial CARDIOLIPIN level. Other biochemical and morphological mitochondrial abnormalities also exist.

Also Known As: MGA Type 2; 3 methylglutaconicaciduria type 2; 3-methylglutaconicaciduria type 2s; 3-methylglutaconicaciduria type IIs; MGA type IIs Show All >>

Networked: 116 relevant articles (3 outcomes, 5 trials/studies) for this Disease

Key Drugs and Agents for Barth Syndrome

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Cardiolipins (Cardiolipin) : 1 outcome 4 studies in 55 results : IBA
  2. Reactive Oxygen Species (Oxygen Radicals) : 1 outcome in 2 results : IBA
  3. TEMPO : 1 outcome in 1 result : IBA
  4. Linoleic Acid (Linoleate) : 1 study in 4 results : IBA
  5. Messenger RNA (mRNA) : 1 study in 3 results : IBA
  6. monolysocardiolipin : 8 results : IBA
  7. Cyclic neutropenia : 5 results : IBA
  8. Proteins (Proteins, Gene) : 4 results : IBA
  9. Cataract and cardiomyopathy : 4 results : IBA
  10. AMP Deaminase (Myoadenylate Deaminase) : 3 results : IBA
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Diseases Related to Barth Syndrome

  1. Neutropenia
  2. Dilated Cardiomyopathy (Cardiomyopathy, Congestive)
  3. Muscular Diseases (Myopathy)
  4. Cardiomyopathies (Cardiomyopathy)
  5. Myotonic Dystrophy (Dystrophia Myotonica)
Show All >>

Key Therapies for Barth Syndrome

Efficacy Chart >>
  1. Heart Transplantation (Grafting, Heart) : 4 results
  2. Mitral Valve Annuloplasty : 1 result
  3. Transplants (Transplant) : 1 result
  4. Transplantation (Transplant Recipients) : 1 result

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