1 Guidance

1 Guidance

1.1 Ataluren, within its marketing authorisation, is recommended for treating Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene in people aged 5 years and older who can walk, only when:

  • the company provides ataluren with the discount agreed in the patient access scheme

  • the conditions under which ataluren is made available are set out in the managed access agreement between the company and NHS England, which should include the conditions set out in sections 5.12–5.15 and 5.23 of this guidance.

1.2 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.