who are suitable to use Translarna with Duchenne

A drug to treat a particular form of Duchenne muscular dystrophy has been given the green light by the European Medicines Agency and could be available in the UK in six months.

Translarna is only relevant to patients with a ‘nonsense mutation’, who make up 10-15% of those affected by Duchenne.

The EMA decided not to pass the drug in January, but they have since re-examined the evidence.

A campaign group said the drug must reach the right children without delay.

There are currently no approved therapies available for this life-threatening condition.

The patients who will benefit the most are those aged five years and over who are still able to walk, the EMA said.
Duchenne muscular dystrophy is a genetic disease that gradually causes weakness and loss of muscle function.

A new drug could be used to treat children in the UK with Duchenne in 2015

A new drug could be used to treat children in the UK with Duchenne in 2015


Patients with the condition lack normal dystrophin, a protein found in muscles, which helps to protect muscles from injury.

In patients with the disease, the muscles become damaged and eventually stop working.

There are 2,400 children in the UK living with muscular dystrophy, but only those whose condition is caused by a particular ‘nonsense mutation’ – namely 200 children – are suitable to use Translarna.

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