The European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion regarding Raptor Pharmaceutical Corporation's drug, Procysbi, for treating proven nephropathic cystinosis, a rare inherited genetic disorder that causes irreversible tissue damage, organ failure and premature death. On September 20th, 2010, Procysbi was designated as an orphan medicinal product in Europe, because it targets a rare condition or disease. Cystinosis is a very uncommon genetic condition that affects approximately 3,000 people worldwide...
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