The first gene therapy to be approved in a regulated market was announced by the European Commission, which granted its approval to a treatment for the rare inherited disorder lipoprotein lipase deficiency (LPL). This genetic disorder leaves people unable to store fat in adipose and muscle cells. The new drug has been named Glybera by its maker, UniQure. The therapy, which manufacturers say will be available next year, uses a virus to insert a working copy of the gene into muscle.
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- Is gene therapy emerging from technological prematurity?
- Gene therapies need new development models
- Gene therapy awaits approval in Europe
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