Alpha-mannosidosis

Living with

How can a stem cell transplant help with the symptoms of Alpha-mannosidosis?

A hematopoietic stem cell transplant, or HSCT, replaces the missing alpha-mannosidase enzyme in people with Alpha-mannosidosis. This can improve some of the symptoms of the Alpha-mannosidosis like a large liver and spleen, frequency of infections, and hearing loss. A HSCT does not usually improve the problems with the bones, problems with the eyes, or the problems with mental development and the brain. Since Alpha-mannosidosis is a progressive condition, having a HSCT is considered to be most beneficial early on in life. HSCT is not done routinely and is a risky procedure. There are some reports of people with Alpha-mannosidosis having successful HCST and other reports of people with this condition not having a successful HCST. HCST has significant risks to the person undergoing the procedure. The risks and benefits of HSCT should be discussed with your doctor.

http://www.ncbi.nlm.nih.gov/books/NBK1396/

http://www.mpssociety.org.uk/files/5313/6914/1863/Guide-AlphaMannosidosis-2013.pdf

http://www.ojrd.com/content/3/1/21

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