Open letter to Sir Nick Partridge Chair of CPAG Nick Catlin Tuesday, 2 December 2014 No Comment

Dear Sir Nick Partridge


I have a son Saul who is now 14 and living with Duchenne Muscular Dystrophy. I still remember holding him as a little baby in a small room when the neuromuscular consultant told us that he had Duchenne. We kind of knew what this would mean. The muscle wasting is severe and now is affecting his everyday life. He told me the other day that any kind of drug would help just to stop him from being so tired everyday. Saul is studying hard for his GCSE’s and to his credit he has overcome some tough steps in learning to read to become a great student. He wants to design computer games or maybe be an archaeologist!


Unfortunately the new drug Translarna will not help him as he does not have the right gene variation for it to work. However this drug can help other young people living with Duchenne in the UK who might just not have to suffer such extreme muscle wasting that my own son has experienced. Also we know that other personalised medicines for Duchenne might be coming on stream through clinical trials currently in progress and so there are real prospects for treating most if not all the boys.


So it is vital that the CPAG committee understand the need to fund Translarna and also other new medicines when they become licensed.


Saul suggested that I set up a petition https://you.38degrees.org.uk/p/translarna and I’m delighted to say that the Duchenne Community and many others (including the dog Elvis!) are supporting our call to fully fund this treatment.

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Translarna and other drugs downstream can offer significant improvement in the quality and length of life for our sons. These drugs can keep young people out of hospital, freeing up beds and saving money in the longer term. However we know that they are still experimental and boys will need long term exposure to monitor efficacy and any side effects.


The only way we can ensure that smaller pharmaceutical companies like PTC Therapeutics stay developing these personalised medicines for rare diseases is if they can see that they will get a reasonable return on their long term investments. So it is crucial that the NHS have a budget that is going to meet the costs of delivering these novel therapies.


Families have been waiting a long time for new treatments to come on stream and everyone in the Duchenne community urges the NHS in England, Scotland, Wales and Northern Ireland to fully fund the costs of Translarna and support its immediate delivery to patients.


Thank you for your support and please can you make sure that your committee members are fully aware of our growing petition now at over 10000 signatures https://you.38degrees.org.uk/p/translarna


Nick Catlin

2nd December 2014

79 is hosted by Nick Catlin

Nick Catlin has a son Saul who has Duchenne Muscular Dystropy. He is a founding member and previous CEO of the Charity Action Duchenne. Nick now works with young people with Duchenne and their families at Decipha assessing for Special Education Needs and advising on reviewing Education Health and Care Plans.

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