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Adult Onset Sandhoff's Disease


  I was diagnosed approx. 2 years ago with Adult Onset Sandhoff's Disease.
  I have been experiencing severe and progressive muscle weakness in my
  legs and am beginning to experience weakness in my arms as well.  I understand what this disorder is and that there is are no known treatments at this time.  I am searching for anyone who has special training in this area
  and who might know of any new therapies being attempted.  I would greatly
  appreciate any information that you could provide.
                                                                    Thank you,
                                                                     Tammy
===========================================================================
Dear Tammy:
Adult-onset Sandhoff's disease (GM2 gangliosidosis with partial deficiency of hexosaminidase-A and -B) is a rare condition. There is no known treatment that arrests or reverses the neurological involvement from this condition. Animal studies of bone marrow transplantation to treat Sandhoff's disease have been recently published ( Norflus F; Tifft CJ; McDonald MP; Goldstein G; Crawley JN; Hoffmann A; Sandhoff K; Suzuki K; Proia RL: Bone marrow transplantation prolongs life span and ameliorates neurologic manifestations in Sandhoff disease mice. J Clin Invest, May 1 1998, 101(9) p1881-8). For further information on this matter, it may be best if you can contact (or have your physician contact) the investigators listed above at the Genetics and Biochemistry Branch of the National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, Maryland.
Good luck!  





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