National Center for Advancing and Translational Sciences Genetic and Rare Diseases Information Center, a program of the National Center for Advancing and Translational Sciences

GM1 gangliosidosis

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

The following information may help to address your question:


How might GM1 gangliosidosis be treated?

There is currently no effective medical treatment for GM1 gangliosidosis.[1][2] 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.[1]

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.[2] 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.[2]

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.[4] 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

We hope this information is helpful. We strongly recommend you discuss this information with your doctor. If you still have questions, please contact us.

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GARD Information Specialist

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  1. NINDS Gangliosidoses Information Page. National Institute of Neurological Disorders and Stroke (NINDS). October 2011; https://www.ninds.nih.gov/Disorders/All-Disorders/Generalized-Gangliosidoses-Information-Page.
  2. Tegay D. GM1 Gangliosidosis. Medscape. March 29, 2012; http://emedicine.medscape.com/article/951637-overview.
  3. GM1 Gangliosidosis - Infantile. Hide & Seek Foundation for Lysosomal Disease Research. http://www.hideandseek.org/Diseases.html.
  4. David H. Tegay. GM1 Gangliosidosis. Medscape Reference. March 29, 2012; http://emedicine.medscape.com/article/951637-overview. Accessed 8/6/2012.