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Niemann-Pick Diseases (Niemann Pick Disease) Summary

Description: A group of autosomal recessive disorders in which harmful quantities of lipids accumulate in the viscera and the central nervous system. They can be caused by deficiencies of enzyme activities (SPHINGOMYELIN PHOSPHODIESTERASE) or defects in intracellular transport, resulting in the accumulation of SPHINGOMYELINS and CHOLESTEROL. There are various subtypes based on their clinical and genetic differences.

Also Known As: Niemann Pick Disease; Niemann Pick Diseases; Niemann-Pick Disease

Networked: 328 relevant articles (6 outcomes, 23 trials/studies) for this Disease

Key Drugs and Agents for Niemann-Pick Diseases

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Sphingomyelin Phosphodiesterase (Sphingomyelinase) : 4 outcomes 7 studies in 153 results : IBA
  2. neodymium pyrocatechin disulfonate (NPD) : 1 outcome 5 studies in 115 results : IBA
  3. Cholesterol : 1 outcome in 33 results : IBA
  4. Intercellular Adhesion Molecule-1 (Intercellular Adhesion Molecule 1) : 1 outcome in 2 results : IBA
  5. Sphingomyelin lipidosis : 3 studies in 34 results : IBA
  6. Lipids : 2 studies in 10 results : IBA
  7. Proteins (Proteins, Gene) : 2 studies in 5 results : IBA
  8. Lipoproteins (Lipoprotein) : 1 study in 6 results : IBA
  9. Enzymes : 1 study in 6 results : IBA
  10. Phosphatidylcholines (Phosphatidylcholine) : 1 study in 4 results : IBA
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Diseases Related to Niemann-Pick Diseases

  1. Central Nervous System Diseases (CNS Diseases)
  2. Gaucher Disease (Gaucher's Disease)
  3. Mucolipidoses (Sialidosis)
  4. Lysosomal Storage Diseases (Lysosomal Storage Disease)
  5. Mucopolysaccharidoses
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Key Therapies for Niemann-Pick Diseases

Efficacy Chart >>
  1. Enzyme Replacement Therapy : 1 outcome in 4 results
  2. Bone Marrow Transplantation (Transplantation, Bone Marrow) : 1 study in 5 results
  3. Subcutaneous Infusions : 1 study in 1 result
  4. Liver Transplantation : 3 results
  5. Stem Cell Transplantation : 2 results
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