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This is my blog where I want to share all that I know about the amazing world of medicine, and biology. The human body is an amazing thing and what it is truly capable of doing.
I will often post blogs on my opinions of medical research and breakthroughs and encourage anybody to comment on it too! Some will be really good and some extremely frustrating!

Saturday, 29 September 2012

Drug Breakthrough-Progeria!

For those who don't know, Progeria is an extremely rare condition found in children, where their body is unable to make a properly functioning type of protein. Instead they make a defective version called 'progerin'. The progerin triggers a range of effects that cause serious damage to parts of the body, mimicking advanced aging.
Symptoms:
Weight loss, hair loss, hardening of the arteries, arthritis, weak bones, and heart disease.

Progeria is often re-labelled as the 'Benjamin Button disease. Given this name due to the novella and film called 'The Curious Case of Benjamin Button' - a child, born old and becomes younger throughout his life.
Researchers experimented with lonafarnib, which was believed to put a stop to some of the effects of progerin protein. It was tested on 26 children affected with the disease. (Quite a large sample, as of how rare this disease is.) One child passed during the course of this experiment from a stroke. The lonafarnib drug however showed promising results as its outcomes were so good. The children experienced improvements in at least one or more of their problems.
To read more about this breakthrough go to the link: http://www.nhs.uk/news/2012/09September/Pages/Drug-breakthrough-for-children-with-premature-ageing-condition.aspx

I think that even though this disease is rare, those few who suffer with it deserve a better way of life. The Lonafarnib drug seems to be a very promising aid. I give credit to the researches and the funding given by the Progeria Research Foundation , the Dana-Farber Cancer Institute and all the other charitable organisations for making this discovery possible.
What lonafarnib is doing for these fighters! In several cases:
Improvement on weight gain.
Improvements of blood vessel stiffness.
Improvements on bone density.
Improvements on hearing loss.


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