What was lorenzos oil why was it so special




















These mono-unsaturated long-chain fatty acids are slightly different than the saturated long chain fatty acids, because they have a single double bond in the fat-chain. It was predicted that the mono-unsaturated fatty acids would compete with the enzymes that are responsible for the elongation of very long-chain fatty acids elongases and that their affinity for mono-unsaturated fatty acids would be higher than for saturated fatty acids. From the initial studies examining the role of this mixture in ALD, it was very apparent that it did not alter the progression of cerebral disease in affected individuals.

This experience has been demonstrated repetitively. In addition, it has never been studied in the preparation for bone marrow transplantation or in the period of time following that treatment.

Initial studies in the adult form of ALD, adrenomyeloneuropathy AMN used the treatment in small populations with variable presentations for short periods of time without definitive effect.

Levels of platelets in the blood are monitored with regular blood tests. Odone A, Odone M. J Pediatr Neurosci. Rizzo, William B. Phillips, Andrea L. Dammann, Robert T. Leshner, Sandra S. Jennings, Joel Avigan, and Virginia K. Moser, Hugo W. Muenz, Ann B. Moser, Jiahong Xu, Richard O. Jones, et al. Miller, W. Others get the adult form of the disease, which strikes people in their late twenties and acts more slowly.

The new study shows that the boys whose parents scrupulously administered the oil — often in the face of scepticism from the medical establishment — were much less likely to go on to develop symptoms in childhood than boys who did not get the oil on a regular basis.

Boys carrying the defect have extremely high levels of very long chain saturated fatty acids in their blood. The oil brings these levels back down to normal in a way that can be tested, and this enabled the researchers to monitor which boys were getting the treatment on a regular basis.

Between and two teams, one led by Moser at the Kennedy Krieger Institute in Baltimore, Maryland, the other based in Europe, looked at the progress of young boys with the defect. At the outset all were under six years of age and none had begun to develop symptoms — their movement, hearing and MRI brain scans were normal.

By the end of the study 76 per cent of the 68 boys getting the oil were still healthy and producing normal brain MRI scans. The same was true of no more than about one in three of the 36 boys who did not regularly get the oil. The big remaining uncertainty is whether those boys in the study who have escaped the childhood version of the disease will also be spared the adult form.



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