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Fabry Disease (Fabry's Disease) Summary

Description: An X-linked inherited metabolic disease caused by a deficiency of lysosomal ALPHA-GALACTOSIDASE A. It is characterized by intralysosomal accumulation of globotriaosylceramide and other GLYCOSPHINGOLIPIDS in blood vessels throughout the body leading to multi-system complications including renal, cardiac, cerebrovascular, and skin disorders.

Also Known As: Fabry's Disease; Anderson Fabry Disease; Disease, Fabry; Angiokeratoma Corporis Diffusum; alpha-Galactosidase A Deficiency Show All >>

Networked: 1607 relevant articles (76 outcomes, 166 trials/studies) for this Disease

Key Drugs and Agents for Fabry Disease

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. agalsidase beta : 12 outcomes 19 studies in 67 results : FDA 2
  2. agalsidase alfa : 10 outcomes 20 studies in 89 results : IBA
  3. alpha-Galactosidase (Beano) : 8 outcomes 37 studies in 688 results : IBA
  4. globotriaosylceramide (globotriosylceramide) : 6 outcomes 7 studies in 213 results : IBA
  5. Biological Markers (Surrogate Marker) : 4 outcomes 5 studies in 32 results : IBA
  6. Galactosidases : 3 outcomes 8 studies in 284 results : IBA
  7. Enzymes : 2 outcomes 2 studies in 26 results : IBA
  8. Lysergic Acid Diethylamide (LSD) : 1 outcome 2 studies in 16 results : IBA
  9. Immunoglobulin G (IgG) : 1 outcome 2 studies in 5 results : IBA
  10. 1-deoxygalactonojirimycin : 1 outcome 1 study in 17 results : IBA
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Diseases Related to Fabry Disease

  1. Pain (Aches)
  2. Gaucher Disease (Gaucher's Disease)
  3. Stroke (Strokes)
  4. Kidney Diseases (Kidney Disease)
  5. Neuralgia (Stump Neuralgia)
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Key Therapies for Fabry Disease

Efficacy Chart >>
  1. Enzyme Replacement Therapy : 40 outcomes 55 studies in 395 results
  2. Intravenous Infusions : 3 outcomes 2 studies in 7 results
  3. Transplantation (Transplant Recipients) : 2 outcomes 1 study in 17 results
  4. Enzyme Therapy : 2 outcomes in 16 results
  5. Bone Marrow Transplantation (Transplantation, Bone Marrow) : 2 outcomes in 3 results
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