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Genetic and Rare Diseases Information Center (GARD)

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GM1 gangliosidosis

Other Names for this Disease
  • Beta galactosidase 1 deficiency
  • Beta-galactosidosis
  • GLB 1 deficiency
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Your Question

Is physical therapy of any benefit to a 6-month-old with GM1?

Our Answer

We have identified the following information that we hope you find helpful. If you still have questions, please contact us.

How might GM1 gangliosidosis be treated?

There is currently no effective medical treatment for GM1 gangliosidosis.[1] Symptomatic treatment for some of the neurologic signs and symptoms is available, but does not significantly alter the progression of the condition.[2][3] For example, anticonvulsants may initially control seizures. Supportive treatments may include proper nutrition and hydration, and keeping the affected individual's airway open.[4]

Bone marrow transplantation was reportedly successful in an individual with infantile/juvenile GM1 gangliosidosis; however, no long-term benefit was reported. Presymptomatic cord-blood hematopoietic stem-cell transplantation has been advocated by some as a possible treatment due to its success in other lysosomal storage disorders.[1] Active research in the areas of enzyme replacement and gene therapy for the condition is ongoing but has not yet advanced to human trials.[2][3]

Neurologic and orthopedic sequelae may prevent adequate physical activity, but affected individuals may benefit from physical and occupational therapy.[1]

Last updated: 8/6/2012

Is physical therapy of any benefit to a 6-month-old with GM1 gangliosidosis?

It has been suggested that affected individuals may benefit from physical and occupational therapy.[1] However, we were unable to locate any studies or reports that discuss the use of physical or occupational therapy for affected individuals in the available literature. Furthermore, we were unable to find information about the type of GM1 gangliosidosis, as well as the age group, for which these types of therapies may be beneficial.
Last updated: 8/6/2012