After several false dawns, the vision of treating debilitating inherited disorders by administering a correct copy of a faulty or missing gene, is about to be realised, following the European Medicines Agency’s (EMA) recommendation that the first such gene therapy Glybera, should receive marketing approval.
This is a triumph for Glybera’s developer, uniQure BV, which has invested €50 million in the treatment for lipoprotein lipase deficiency, an ultra-rare inherited disease in which patients are unable to produce...
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