Sandra suffers from Metachromatic Leukodystrophy (MLD), in which a genetic lack of a particular enzyme leads to a build-up of waste products in nerve cells, destroying their protective sheath. The most common form of the disease emerges in late infancy.

There is currently no cure, but Massart, who lives in Temse, East Flanders, saw promise in the drug Metazym, produced by a small Danish company, Zymenex, which was undergoing clinical trials at the time. The company promised to let him have the drug providing he could pay for it. Thanks to his fund-raising efforts and the generosity of many ordinary people in Belgium, he raised the money, but by that time Zymenex had sold the patent and the drug to Shire, a pharmaceutical giant based in Basingstoke, England.

Shire refused to sell Massart the drugs, claiming their production was only enough for the 12 children involved in the official trial. But they promised to review their decision later. Last week, the Massart family was informed that Shire was stopping development of the drug because of disappointing test results. Metazym was not effective enough, said a company spokesman.

Massart has now turned his attention to the US, where a new treatment has shown promise in not only arresting the progress of MLD, but reversing some effects. Sandra is on the waiting list and now awaits the approval of the Food and Drug Administration for the therapy to go ahead.

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