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Prognosis: Prognosis for patients with the childhood form of ALD is generally poor because it is a quickly progressive disease that leads to profound disability, and death may occur within one to ten years after the onset of symptoms. The neonatal form of ALD also progresses rapidly and can result in death. The later onset forms are significantly less dangerous and usually don't result in death. .
Treatments: Although there is no cure for ALD but there are a few treatments that may halt or even reverse the disease. One successful treatment for the childhood form is a bone marrow transplant. This is most effective with patients who have very little neurological involvement. Unfortunately, newly diagnosed patients are usually too far along in the neurological degradation in order for the bone marrow transplant to be of use. .
In a bone marrow transplant, the bone marrow cells are taken from someone else other than the afflicted individual. A potion of the donated cells, which are not genetically defective, find their way into the brain, express the appropriate ALD protein that had been missing or not functioning properly and the brain damage is halted or reversed. However, it is important that the donor and the patient meet a certain criteria for a successful outcome. With this said, even under the best conditions, bone marrow transplants are very dangerous procedures that have up to a 25% mortality rate. Part of the reason for a high mortality rate is so high is that patients must receive high doses of chemotherapy and/or radiation so that the patients" body will become immunosupressed. This leaves the body immune system at high risk of contracting any infection. In addition, the donor material may recognize the host as foreign, and attack the host. If that was to happen it could lead the point of death. .
Another treatment is a gene therapy bone marrow transplant. This would involve temporarily removing the appropriate cells from the ALD patient, correcting the genetic sequence within these cells so that is would now include the appropriate "blueprints," and putting these cells back into the patient.
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