Get involved Campaign for change Action Duchenne working with Scottish Family to get Translarna approved by SMC Ross Munro, living with Duchenne, was first enrolled on the PTC Therapeutics trial of Translarna in 2008, and has been in receipt of the groundbreaking treatment since the following year. Over that time Ross has experienced the benefits that Translarna can bestow, remaining ambulant and witnessing improvements in his handwriting owed to increased muscle function. Action Duchenne are now working with Ross and his father Kenny to inform the Scottish Medicines Consortium (SMC) evaluation of Translarna, and will be accompanying Kenny to the upcoming Patient and Clinician Engagement (PACE) meeting to make a direct case to the committee for Translarna’s approval. The conclusions of this meeting, taking place on 9 February, will be considered in partnership with the Patient Group Submission Form we submitted at the end of last year, with a decision on Translarna expected in Scotland in the Spring. We are delighted that MDUK and Harrisons Fund, along with parents Gary Fegan and Michelle Young, are also working alongside us to inform this process. We would also, as always, like to thank our Scottish Advocate John Miller for his ongoing support. We believe that being on Translarna has changed the course of Ross’ condition, allowing him to enjoy a relatively independent life for longer. The drug needs to be approved as soon as possible to allow more nmDMD boys in the UK to benefit. - Kenny Munro Action Duchenne will continue to do all we can to ensure that Translarna receives approval all over the UK, and boys eligible for this groundbreaking treatment can benefit as quickly as possible.