NICE recommend drug trialled at GOSH for the treatment of Duchenne Muscular Dystrophy

NICE have published final guidance recommending ataluren for the treatment of Duchenne Muscular Dystrophy (DMD) caused by a nonsense mutation, in children aged 5 years and over who are able to walk.

DMD is a severe, muscle-wasting condition caused by a fault in a gene, resulting in a failure to produce functional dystrophin; currently DMD has few treatment options.

Ataluren is a novel drug, aimed at the root cause of the disease, allowing the body to continue to produce dystrophin by reading over the mutation in the DNA. Ataluren targets a specific change in the DNA called a nonsense mutation, which affects approximately 13% of Duchenne sufferers. Research in which GOSH recruited a substantial number of patients, has suggested that ataluren could delay the loss of walking in these patients by up to 7 years.

A 5-year managed access agreement for ataluren has been arranged, permitting patients with this disorder to access the drug whilst allowing more data to be collected on the drug’s efficacy.

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