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GM1 Gangliosidosis (Gangliosidosis GM1) Summary

Description: An autosomal recessive neurodegenerative disorder caused by the absence or deficiency of BETA-GALACTOSIDASE. It is characterized by intralysosomal accumulation of G(M1) GANGLIOSIDE and oligosaccharides, primarily in neurons of the central nervous system. The infantile form is characterized by MUSCLE HYPOTONIA, poor psychomotor development, HIRSUTISM, hepatosplenomegaly, and facial abnormalities. The juvenile form features HYPERACUSIS; SEIZURES; and psychomotor retardation. The adult form features progressive DEMENTIA; ATAXIA; and MUSCLE SPASTICITY. (From Menkes, Textbook of Child Neurology, 5th ed, pp96-7)

Also Known As: Gangliosidosis GM1; Gangliosidosis, GM1; Adult GM1 Gangliosidosis; Beta Galactosidase 1 Deficiency; Beta-Galactosidase-1 Deficiency Disease Show All >>

Networked: 243 relevant articles (3 outcomes, 16 trials/studies) for this Disease

Key Drugs and Agents for GM1 Gangliosidosis

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Oligosaccharides : 1 outcome 1 study in 14 results : IBA
  2. Biological Markers (Surrogate Marker) : 1 outcome 1 study in 1 result : IBA
  3. Protons (Proton) : 1 outcome in 1 result : IBA
  4. beta-Galactosidase (Lactaid) : 6 studies in 84 results : IBA
  5. N-octyl-beta-valienamine : 2 studies in 4 results : IBA
  6. acid beta-galactosidase : 1 study in 24 results : IBA
  7. Neuraminidase deficiency with beta-galactosidase deficiency : 1 study in 14 results : IBA
  8. Galactose (Galactopyranose) : 1 study in 7 results : FDA 3 Generic
  9. Cholesterol : 1 study in 7 results : IBA
  10. N-Acetylneuraminic Acid (Sialic Acid) : 1 study in 4 results : IBA
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Diseases Related to GM1 Gangliosidosis

  1. GM1 Gangliosidosis (Gangliosidosis GM1)
  2. Mucolipidoses (Sialidosis)
  3. Mucopolysaccharidosis IV (Morquio Syndrome)
  4. Globoid Cell Leukodystrophy (Krabbe Disease)
  5. Fabry Disease (Fabry's Disease)
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Key Therapies for GM1 Gangliosidosis

Efficacy Chart >>
  1. Enzyme Replacement Therapy : 1 study in 1 result
  2. Lasers (Laser) : 1 study in 1 result
  3. Enzyme Therapy : 1 result
  4. Cell Transplantation : 1 result
  5. Tissue Transplantation : 1 result
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