Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene | DuchenneXchange

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Guides & Guidelines

Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene

key information

source: National Institute for Health and Care Excellence

year: 2016

summary/abstract:

This guidance is not intended to affect the position of patients whose treatment with ataluren was started within the NHS before this guidance was published. Treatment of those patients may continue without change to whatever funding arrangements were in place for them before this guidance was published until they and their NHS clinician consider it appropriate to stop.

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