APPROVAL FOR DUCHENNE MUSCULAR DYSTROPHY TREATMENT, TRANSLARNA

Motion Text

That this House welcomes the decision by the National Institute for Health and Care Excellence (NICE) to recommend the Duchenne muscular dystrophy treatment, Translarna, for funding by NHS England, as a result of a Managed Access Agreement; notes that Translarna is the first ever approved drug to treat an underlying genetic cause of Duchenne muscular dystrophy; recognises the tireless campaigning by patients and families throughout the NHS England and NICE processes calling for access to Translarna; acknowledges the vital role that Muscular Dystrophy UK and Action Duchenne have played as patient organisation stakeholders during the NICE assessment process; further recognises the role of other charities in pressing for access to Translarna; further notes with concern the announcement by the Scottish Medicines Consortium to recommend that Translarna not be approved for funding by NHS Scotland; and calls on NHS England to shorten implementation from three months so that the drug is available as quickly as possible.