I Am Writing to You Regarding Access to Translarna, a New Treatment for Duchenne Muscular

Dear

I am writing to you regarding access to Translarna, a new treatment for Duchenne muscular dystrophy.

The Scottish Medicines Consortium has announced that it will not be recommending the drug for funding on the NHS. This is a major blow for families, who have waited for a decision on funding since the drug received a license from the European Medicines Agency in August 2014. Translarna is the only licensed drug to address an underlying genetic cause of Duchenne, so there are no alternative drug treatment options.

Families have since learned that the drug is to approved in England under a Managed Access Agreement, meaning that children are expected to have access to the drug on the NHS in England from this summer.

Duchenne muscular dystrophy, which almost exclusively affects boys, is a severe muscle-wasting condition caused by the lack of a muscle protein called dystrophin.The condition affects approximately 2,500 individuals in the UK, with around 100 new cases diagnosed each year.Eventually, the progression of the condition leads to serious muscle wasting in the heart and the lungs. Currently, few of those with the condition will live to see their thirtieth birthday.

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Translarna has been shown in clinical trial to slow the decline of Duchenne muscular dystrophy. It would therefore keep boys walking for longer, delay the need for a wheelchair and potentially delay the onset devastating cardiac and respiratory weakness which comes in the later stages of the condition. Eligible boys in countries including Germany, France, Italy, Spain, Greece, Austria, Norway, Sweden, Denmark, Slovakia have already had the treatment approved for use.

The SMC has said that they are concerned at the price of the drug, and a meeting is set between the SMC and the drug company PTC Therapeutics very soon.

Everyone involved has a duty to make sure we get the best outcome for children like Michael who have Duchenne.

I am therefore writing to ask if you will write to the First Minister of Scotland, Nicola Sturgeon, asking her to intervene and promote a Managed Access Agreement between the drug company, the SMC and NHS Scotland. This will allow children access to the drug at a reduced cost to the NHS and enable longer term evidence to be gathered on the drug’s use.

I hope you’ll be able to show your commitment to enabling access to emerging treatments for individuals in Scotland living with devastating conditions such as Duchenne muscular dystrophy.