rare genetic disease

Glybera gene therapy in approval process in Europe

The European Medicines Agency's Committee for Medicinal Products for Human Use has considered the use of Glybera to treat lipoprotein lipase deficiency

Europe is on the process of approving a gene therapy for the first time, in what would be a landmark moment for the field. Gene therapies alter a patient’s DNA to treat inherited diseases passed from parent to child. The European Medicines Agency has recommended a therapy for a rare genetic disease which leaves people [...]

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