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June 24, 2014
07:07 EDTRPTPRaptor Pharmaceuticals announces publication of PROCYSBI data
Raptor Pharmaceutical announced that data from its open label extension study of delayed-release cysteamine bitartrate, or PROCYSBI, has been published in the Journal of Pediatrics. The study demonstrated that patients with nephropathic cystinosis who took PROCYSBI for two years were able to sustain optimal cystine control in their white blood cells and preserve kidney function over the long term, and had significant improvements in social, school and total functioning based on validated quality of life measurement scales. There were no unexpected or serious safety concerns attributable to PROCYSBI. All 40 patients experienced one or more treatment-emergent adverse events.
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