Tay-Sachs Disease Treatment Research Papers
Tay-Sachs Disease Treatment projects focus on the various medical treatments for the disease Tay-Sachs. Medical writers with a background in Treatment and rehabilitation write on the steps required for treating Tay-Sachs or any other topic you never covered regarding the disease.
According to most sources, once a child develops symptoms there is no known treatment for classic Tay-Sachs disease. The prognosis, therefore, is for a rapidly declining condition that results in inevitable death. Therefore, the best one can do is comfort the child and care for its physical requirements. In many respects, hospice care is appropriate for these children.
However, there is hope for some treatments.
- A study presented at the 2000 Pediatric Academic Societies and American Academy of Pediatrics Joint Meeting reported that safe transfer of healthy genes in the womb may help treat genetic diseases such as Tay-Sachs disease.
- A pre-clinical study safely transferred healthy genes to animal fetuses in the womb by transferring the healthy genetic material through a "retroviral vector system," or non-disease causing virus, directly to the damaged cells.
- The results, which were seen through gestation and after the fetus was born, could treat genetic disorders, such as Tay-Sachs, in the womb and eliminate damage that occurs at onset.
There are currently compounds being developed to treat lysosomal storage disorders such as Tay-Sachs. These drugs are designed to inhibit the production of glycolipids, the fatty substances that accumulate abnormally in Tay-Sachs patients. Research paper writers report a similar strategy for the treatment of Tay-Sachs, based on an inhibitor of glycolipids, was tested in a mouse model of Tay-Sachs disease. When Tay-Sachs mice were treated with N-butyldeoxynojirimycin, the accumulation of fatty substances in the brain was prevented, with the number of storage neurons and the quantity of ganglioside stored per cell reduced. In addition to preventing an imbalance of fatty substance, the neuropathology associated with this disorder was also prevented. These new approaches to treatment of Tay-Sachs offers hope to many families who have no treatment for their dying children. However, side effects of these drugs may be severe. No mortality rates for animal tests and no human test results were available at this time.