I have no clue how old this info is (the website says 1997), but it is worth looking into seeing how this panned out…
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In the past couple of years, attempts to treat Type 2 babies with Ceredase have been made in one case in America by Prof Gregory Grabowski, Director of Genetics, Children’s Hospital, Cincinatti, and in two cases in Italy by Dr Bruno Bembi at the Children’s Hospital, Trieste, but without success.
Work is also being carried out in America on a drug called L-cycloserine which is claimed to help Type 2 children. Experiments have taken place on ‘mice with Gauchers’ and the drug has been in use on humans suffering from other illnesses, such as tuberculosis, for some time. So far it has not been used on Type 2 babies but Dr Meir Lev, Director of Research Services and Development, City University of New York Medical School, believes it could be effective. This drug works by slowing down the accumulation of Gaucher cells in the body rather than supplementing the missing enzyme (which is what Ceredase does). The theory is by preventing new accumulation, any residual enzyme produced in the body will reduce or eventually eliminate the existing Gauchers cells.
Similar research is being carried out with a different substance called NB-DNJ by Dr Terry Butters and Dr Frances Platt at the Glycobiology Institute, University of Oxford. It is hoped that this or a similar substance will also inhibit the accumulation of Gauchers cells.
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More things to check out:
http://www.investmentguide.co.uk/gaucher/dec2007p13.pdf
http://www.investmentguide.co.uk/gaucher/contents.htm
Ok, how do we get ahold of these doctors?
It all helps the body but not the brain. They have yet to find something that crosses the blood to brain barrier.