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Thursday, July 14, 2011

Transplant Med may help kids with rare disease

:Original raster version: :Image:Food and Drug...Researchers may have found another use for the anti-rejection medication Rapamycin. A study has shown that the drug could be used to treat a very rare childhood disease called Hutchinson-Gilford progeria syndrome (HGPS). The disease is very severe, but is so rare that since it's discovery in the early 1900s, only 100 cases have been diagnosed.

HGPS basically causes children to age too quickly. It causes them to develop joint disease, skin issues, and severe cardiovascular disease generally killing its victims in their teens. The disease is caused by the body producing too much of a protein called progerin. Progerin is present in everyone in very small amounts. However, as we age, it accumulates in our bodies causing cell damage leading to the typical symptoms of old age.

Rapamycin, though, has been shown to reverse the damage caused in children with HGPS and may be the answer that will keep them from growing old before their time. Plus, since Rapamycin is already on the market, it's safety record and side effects are known, and the FDA has approved it's use, it should be available for treatment of HGPS fairly quickly.

Personally, I think it's neat that a drug used in organ transplantation has now been shown to have other beneficial uses. Plus, here's a thought - since the drug appears to reverse the aging process, maybe at some point in the future this medication, or one similar, will benefit the rest of us, too.

Source : health.usnews.com article titled Organ Transplant Drug Might Treat Rapid Aging Disease in Kids



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