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Research Article – Natural History of Infantile GM2 Gangliosidosis

In November 2011 a research article called “Natural History of Infantile GM2 Gangliosidosis” was published in the Official Journal of the American Academy of Pediatrics. The article was written by Annette E. Bley, Ourania A. Giannikopoulos, Doug Hayden, Kim Kubilus, Cynthia J. Tifft and Florian S. Eichler.

The research article

The objective of the study looked at trying to quantify the rate of functional decline in infantile GM2 gangliosidosis on the basis of patient surveys and a comprehensive review of existing literature (Bley et al, 2011).

You can download the research article by clicking on the image below or you can access an online version here.

Natural History of Infantile GM2 Gangliosidosis

 
By studying the timing of regression in 97 cases of infantile GM2 gangliosidosis Bley et al (2011) are able to conclude that clinical disease progression does not correlate with survival, likely because of the impact of improved supportive care over time.
 
Source: Annette E. Bley, Ourania A. Giannikopoulos, Doug Hayden, Kim Kubilus, Cynthia J. Tifft and Florian S. Eichler (2011), Natural History of Infantile GM2 Gangliosidosis, Pediatrics 2011;128;e1233; originally published online October 24, 2011; DOI: 10.1542/peds.2011-0078

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