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Still, getting orphan status is nothing to sneeze at. It is important, especially for small companies. In this article, there is one orphan indication I would like to discuss specifically as far as recent developments are concerned, christian louboutin shoes sale
and that is retinitis pigmentosa.
Retinitis pigmentosa is a genetic disorder, usually autosomal dominant, where the rods and cones in the back of the eye on the retina die due to malfunctioning genes. The cause can be any number of different mutations on key genes responsible for producing proteins that protect these cells. The result is tunnel vision christian louboutin on sale real
and eventually blindness by adulthood. The fact that it is usually dominant means that the chances of inheriting it from a parent are christian louboutin online sale
at least 50%.
RP is not a disease one generally hears much about. One telling statistic is that there are only that even mention it on Seeking Alpha. But the patient base is not insignificant. Globally, it is in the median range for an orphan 100,000 people in the US, another 100,000 in Europe, and 50,000 in Japan will end up blind from this disease, christian louboutin sale mens most of them by the age of 40.
Currently there are 3 publicly traded companies actively pursuing a treatment for RP, all in different stages of clinical development. Let us begin with the farthest down the line in phase 3 and that is Sucampo Pharmaceuticals (NASDAQ:SCMP), partnered with the Japanese biotech R Tech Ueno.
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