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Mucolipidoses (Sialidosis) Summary

Description: A group of inherited metabolic diseases characterized by the accumulation of excessive amounts of acid mucopolysaccharides, sphingolipids, and/or glycolipids in visceral and mesenchymal cells. Abnormal amounts of sphingolipids or glycolipids are present in neural tissue. Mental retardation and skeletal changes, most notably dysostosis multiplex, occur frequently. (From Joynt, Clinical Neurology, 1992, Ch56, pp36-7)

Also Known As: Sialidosis; I-Cell Disease; Mucolipidosis; Pseudo-Hurler Polydystrophy; Mucolipidosis I Show All >>

Networked: 587 relevant articles (8 outcomes, 36 trials/studies) for this Disease

Key Drugs and Agents for Mucolipidoses

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Neuraminidase (Sialidase) : 2 outcomes 10 studies in 104 results : IBA
  2. Enzymes : 1 outcome 4 studies in 79 results : IBA
  3. Neuraminidase 1 deficiency : 1 outcome 3 studies in 51 results : IBA
  4. N-Acetylneuraminic Acid (Sialic Acid) : 1 outcome 2 studies in 29 results : IBA
  5. Neuraminidase deficiency with beta-galactosidase deficiency : 1 outcome 1 study in 53 results : IBA
  6. Hydrolases : 1 outcome 1 study in 46 results : IBA
  7. G(M3) Ganglioside : 1 outcome in 5 results : IBA
  8. Cystine (L-Cystine) : 1 outcome in 4 results : IBA
  9. 5-Hydroxytryptophan (5 Hydroxytryptophan) : 1 outcome in 1 result : IBA
  10. beta-Galactosidase (Lactaid) : 3 studies in 36 results : IBA
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Diseases Related to Mucolipidoses

  1. Mucolipidoses (Sialidosis)
  2. Lysosomal Storage Diseases (Lysosomal Storage Disease)
  3. Mucopolysaccharidoses
  4. GM1 Gangliosidosis (Gangliosidosis GM1)
  5. Gaucher Disease (Gaucher's Disease)
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Key Therapies for Mucolipidoses

Efficacy Chart >>
  1. Bone Marrow Transplantation (Transplantation, Bone Marrow) : 1 outcome in 3 results
  2. Continuous Positive Airway Pressure : 1 outcome in 1 result
  3. Anesthesia : 3 results
  4. Aftercare (After-Treatment) : 3 results
  5. Transplants (Transplant) : 2 results
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