Published: 26 July 2012
Breakthrough for Europe as EMA gives the nod to the first gene therapy
Nuala Moran, Science|Business
At the fourth time of asking, the European Medicines Agency is recommending approval of Glybera, opening up a new era in the treatment of rare diseases, and unlocking the wider potential of gene therapy
Image: Bigstock
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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