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... $2 this year, mostly on a July announcement of positive trial results for a new treatment for lupus, an autoimmune disease that causes inflammation and tissue damage. Stock screening software isn't useful for sorting biotechs by the value of their ...
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... will target. So-called orphan medicines currently exist to treat illnesses including the genetic disorder Gaucher disease . His comments about acquiring new treatments and building an orphan-drug business underscore the pressure facing drugmakers , ...
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... neurological disorders called lysosomal storage diseases, the UI team found that these diseases cause unique and disease-specific alterations to the blood vessels of the blood brain barrier. The scientists used these distinct alterations to target ...
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... pivotal study for its lead product candidate, UPLYSO (prGCD), to be used in enzyme replacement therapy for Gaucher disease, a rare and serious lysosomal storage disorder in humans with severe and debilitating symptoms. Protalix and the U.S. Food and ...
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... the diagnostic industry. Many specialist therapies are very costly (e.g. almost $300,000 annually for Fabry's or Gaucher's disease) and are used to treat smaller target patient populations with specific disease subtypes. In this context, there is ...
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... Company's proprietary plant-cell expressed recombinant form of glucocerebrosidase (GCD) for the treatment of Gaucher disease. The trial enrolled a total of 31 patients across Europe, North America, South America, Israel and South Africa. No serious ...
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