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Glycogen Storage Disease Type II (Pompe's Disease) Summary

Description: An autosomal recessively inherited glycogen storage disease caused by GLUCAN 1,4-ALPHA-GLUCOSIDASE deficiency. Large amounts of GLYCOGEN accumulate in the LYSOSOMES of skeletal muscle (MUSCLE, SKELETAL); HEART; LIVER; SPINAL CORD; and BRAIN. Three forms have been described: infantile, childhood, and adult. The infantile form is fatal in infancy and presents with hypotonia and a hypertrophic cardiomyopathy (CARDIOMYOPATHY, HYPERTROPHIC). The childhood form usually presents in the second year of life with proximal weakness and respiratory symptoms. The adult form consists of a slowly progressive proximal myopathy. (From Muscle Nerve 1995;3:S61-9; Menkes, Textbook of Child Neurology, 5th ed, pp73-4)

Also Known As: Pompe's Disease; Glycogenosis Type II; Acid Maltase Deficiency; Adult Glycogen Storage Disease Type II; Alpha-1,4-Glucosidase Deficiency Show All >>

Networked: 919 relevant articles (66 outcomes, 87 trials/studies) for this Disease

Key Drugs and Agents for Glycogen Storage Disease Type II

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. human GAA protein (Myozyme) : 19 outcomes 16 studies in 87 results : FDA 3
  2. alpha-Glucosidases (Acid Maltase) : 11 outcomes 22 studies in 403 results : IBA
  3. Glucosidases : 6 outcomes 6 studies in 159 results : IBA
  4. Glycogen : 5 outcomes 6 studies in 254 results : IBA
  5. Antibodies : 4 outcomes 3 studies in 19 results : IBA
  6. Enzymes : 2 outcomes 2 studies in 18 results : IBA
  7. Acarbose : 2 outcomes 1 study in 8 results : FDA 4 Generic
  8. IGF Type 2 Receptor (Insulin-Like-Growth-Factor II Receptor) : 2 outcomes in 5 results : IBA
  9. bortezomib (Velcade) : 2 outcomes in 2 results : FDA 1
  10. Periodic Acid : 1 outcome in 3 results : IBA
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Diseases Related to Glycogen Storage Disease Type II

  1. Glycogen Storage Disease Type II (Pompe's Disease)
  2. Cardiomyopathies (Cardiomyopathy)
  3. Fabry Disease (Fabry's Disease)
  4. Gaucher Disease (Gaucher's Disease)
  5. Glycogen Storage Disease (Glycogenosis)
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Key Therapies for Glycogen Storage Disease Type II

Efficacy Chart >>
  1. Enzyme Replacement Therapy : 33 outcomes 32 studies in 241 results
  2. Enzyme Therapy : 3 outcomes 5 studies in 20 results
  3. Mechanical Ventilators (Ventilator) : 3 outcomes in 8 results
  4. Anesthesia : 1 outcome in 5 results
  5. Artificial Respiration (Mechanical Ventilation) : 1 outcome in 4 results
Show All >>

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