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Drug to treat Duchenne muscular dystrophy is cleared by EU

By Amanda Stewart,

European Union regulators have given approval for a new drug that will treat Duchenne muscular dystrophy in children.

The drug, Translarna, treats muscular dystrophy by helping the body create a protein called dystrophin, according to Tech Times. This protein helps protect against muscle impairment.

Those who will benefit from the release of the drug will be children ages five and up who are still able to walk.

The decision has been called “historic” because of the number of people that it will help. It was allowed under an exemption that allows a drug to be used when there are no other treatment options.

Presently, there are no treatments for the disease. It primarily affects boys and is a result of a gene mutation that ends the production of dystrophin.
Most of those diagnosed with Duchenne muscular dystrophy are diagnosed before the age of five, using a powered wheelchair by 12, can’t pick up a glass of water by the age of 20 and are usually dead before their 30th birthday.

The EU will allow the drug to be readily available in the 28 countries that are a part of the EU.

According to The Utah Peoples Post, the drug is supposed to become available in the UK in six months.

“This step forward for Translarna offers much needed hope," Chief executive of the campaign, Robert Meadowcraft said.

 

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