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European regulators back hemophilia gene therapy

December 16, 2022

The agency that recommends what drugs should be approved in Europe has thrown its support behind five new ones, including a gene therapy for hemophilia, a combination treatment for liver cancer and a medicine for a rare disorder known as Pompe disease.

While the European Commission still needs to authorize these treatments, the nods from the European Medicines Agency and its drug review committee put them a step closer to market.

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