https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/brc-news/nice-recommend-drug-trialled-gosh-treatment-duchenne-muscular-dystrophy/
NICE recommend drug trialled at GOSH for the treatment of Duchenne Muscular Dystrophy
16 Sep 2016, 11:02 a.m.
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.
Read full article here.
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