"Extraordinary Measures" a Film Inspired by a Drug for Rare Disease in Kids
"Extraordinary Measures" a Film Inspired by a Drug for Rare Disease in Kids

Being only 5 years old, Clay Bowden isn't known to the fact of the drama besieging the drug that keeps him alive.

"Extraordinary Measures," starring Harrison Ford and Brendan Fraser, depicts a fictionalized account of how a real drug came to market in 2006 and saved hundreds of youngsters, including Bowden, suffering with a rare metabolic disorder called Pompe disease.

It is revealed that the movie has some massive ties with Duke University, where that treatment was developed.

"This is an incredible experience. It's very humbling experience for me as a treating physician", said Dr. Priya Kishnani. "It actually reminds me each and every day how precious life is".

That treatment replaces an enzyme that people with Pompe disease do not produce at all, or at severely low a level, which prevents the development of a starch called glycogen, much like insulin prevents the build-up of glucose. Glycogen accumulation results in muscles degradation.

In 1999, Duke began clinical trials in humans. Three children were involved; one responded well and the other two eventually died. Given the lethal nature of Pompe, the results were a huge improvement.

The third and largest clinical trial witnessed the strongest results, and the Food and Drug Administration granted approval for the drug, called Myozyme, in 2006.

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