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Gene therapy: Glybera approved by European Commission
- Author, James Gallagher
- Role, Health and science reporter, 成人快手 News
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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