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Back in 1994, the literature available on Niemann Pick Disease was very scarce and not reassuring. We were told that her life expectancy would be into early childhood. We were then given the option to try a bone marrow transplant, if it works it could give her the enzyme that she needs to survive. It has never been done for this disease in the United States, but tried once in Europe without positive results. We knew that even though we were fortunate to have found a donor in the family, her chances of surviving a bone marrow transplant was not very promising.
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By the age of two, Cassie had already endured two bone marrow transplants. The procedures consisted of many risky treatments used with a cancer diagnosis, which involved the use of radiation, chemotherapy, transfusions and many other drugs accompanied by lengthy hospital stays.
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The bone marrow transplants have given Cassie the enzyme required to cure her of Niemann Pick Disease, however, it truly came at a very high price. Cassie now has to live with chronic graft vs host disease, liver fibrosis, renal tubular acidosis, growth deficiency, multiple contractures of the joints, osteoporosis, and cataracts.
There is a promising new cure for children and adults with Niemann Pick Type B Disease. Enzyme replacement therapy will be available soon, and no one will have to suffer through the treatments and serious side effects that Cassie has endured.
We are very grateful to the medical communities and foundations that have given us valuable support to help us live with Niemann Pick Disease.
Click on the link below and go to the Niemann Pick Web site to read about this devastating disease that robs our children of their childhood. Please help us get closer to a cure, donate to the Niemann Pick Foundation.
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