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Gene therapy: Glybera approved by European Commission

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Gene therapies alter a patient's DNA

A treatment which corrects errors in a person's genetic code has been approved for commercial use in Europe for the first time.

The European Commission has given Glybera , meaning it can be sold throughout the EU.

It is a gene therapy for a rare disease which leaves people unable to properly digest fats.

The manufacturers say it will be available next year.

Gene therapy has a simple premise. If there is a problem with part of a patient's genetic code then change the code.

However, the field has been plagued with problems. Patients have developed leukaemia and in one trial in the US a teenager died.

In Europe and the US, the therapies are used only in research labs.

Fat problems

Glybera is used to treat lipoprotein lipase deficiency. One in a million people have damaged copies of a gene which is essential for breaking down fats.

It means fat builds up in the blood leading to abdominal pain and life-threatening inflammation of the pancreas (pancreatitis).

The only way to manage the condition is by having a very low-fat diet.

The therapy, developed by UniQure, uses a virus to infect muscle cells with a working copy of the gene.

the therapy was made available for the most severely ill patients earlier in 2012.

UniQure chief executive officer Jorn Aldag said: "The final approval of Glybera from the European Commission marks a major step forward in making gene therapies available not only for lipoprotein lipase deficiency, but also for a large number of rare diseases with a very high unmet medical need."

The company said it would apply for regulatory approval in the US and Canada.

China was the first country to .

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