Searching for a cure, Improving lives


Duchenne Research Updates

Posted on: April 26th, 2017 | 0 comments

  This week has seen a number of updates  from organisations working in Duchenne muscular dystrophy research: As always, the best place in the UK to get updates on Duchenne research, is at the annual Action Duchenne International Conference 2017. Find out more, and register here.   Daiichi Sankyo Announces Designation of Duchenne Treatment DS-5141

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BLOG: Being disabled in Britain

Posted on: April 25th, 2017 | 0 comments

Being disabled in Britain, A journey less equal was published by the Equality and Human Rights Commission in April 2017.  It made the headlines this month but its findings are unlikely to be news to families affected by Duchenne Muscular Dystrophy.   The Equality and Human Rights Commission is the national equality body for Great

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Implementation of the Accelerated Access Review

Posted on: April 24th, 2017 | 0 comments

Last week Action Duchenne became members of National Voices, the coalition of charities that stands for people being in control of their health and care. This means we are able to work with many other patient led organisations in the goals we share of ensuring the best outcomes for patients. During a meeting organised by

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Theresa May calls a General Election for June 8 2017

Posted on: April 19th, 2017 | 0 comments

Yesterday the Prime Minister Theresa May called a UK-wide General Election to take place on Thursday 8 June 2017. Further details about the announcement can be found here: We will be providing updates on the whole process of the General Election, key asks from the Duchenne community for prospective parliamentary candidates across the UK

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Bristol-Myers Squibb enters into agreement to license potential Duchenne drug, to Roche

Posted on: April 19th, 2017 | 0 comments

Bristol-Myers Squibb  has entered into an agreement to license BMS-986089, an anti-myostatin adnectin in development for Duchenne Muscular Dystrophy (DMD), to Roche.   “Licensing these assets to…Roche will enable Bristol-Myers Squibb to prioritize the other promising opportunities for asset development that have advanced across our diversified portfolio,” said Mike Burgess, head of Cardiovascular, Fibrosis and Immunoscience

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Eteplirsen to be evaluated by NICE from October 2017

Posted on: April 10th, 2017 | 0 comments

Announcement today Today we have been informed by NICE that the UK’s Department of Health has asked NICE to carry out a Highly Specialised Technology (HST) evaluation of Exondys 51 (eteplirsen) as part of NICE’s programme of evaluating treatments and medicines for Duchenne muscular dystrophy for national commissioning by NHS England. This evaluation will begin

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SKIP-NMD (Skipping exon 53 of the dystrophin gene) Project Update

Posted on: April 10th, 2017 | 0 comments

The aim of the SKIP-NMD project is to develop a potential treatment for Duchenne Muscular Dystrophy by designing and testing a drug developed by Sarepta Therapeutics to restore dystrophin production in young people living with Duchenne who are amenable to exon 53 skipping, missing sections (or “exons”) in the dystrophin gene such as: exons 52, 45-52, 47-52, 48-52,

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Gregor Watson’s Canal Walking Challenge – 98 Miles in 7 Days

Posted on: April 10th, 2017 | 0 comments

  From 8-14 April Gregor Watson, who’s just 13 years old, walked the Cheshire Ring Canal – that’s 98 miles in seven days! Gregor is raising funds for Action Duchenne because his life-long friend Toby Maurice has Duchenne muscular dystrophy, below he explains more about his motivation and what he has been doing to fundraise:  

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Vision-DMD Workshop Videos on Standards of Care Available

Posted on: April 7th, 2017 | 0 comments

United Parents Project Muscular Dystrophy (UPPMD) have published an article and videos from the Vision-DMD – Workshop on Standards of Care, held in Rome in February. These include a summary of the updated standards of care guidelines, presented by experts, that are due to released by the end of the year. Vision-DMD project will undertake

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The Impact of Translarna on our son and our family

Posted on: April 6th, 2017 | 0 comments

In 2008 our family was devastated by our 4-year-old son Isaac’s diagnosis with Duchenne muscular dystrophy.  We found out about Ataluren (translarna) shortly after diagnosis and then waited five years before Isaac was able to take part in the Phase 3 trial.  It was an agonising wait, watching our son deteriorate and fearing that he

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Summit to Extend Ongoing PhaseOut DMD Clinical Trial of Ezutromid in Patients with DMD

March 27th, 2017

  Introduction from Michelle Avery (Summit Therapeutics): Today, we’ve announced that we intend to proceed

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Amazing London Marathon Dinner at Jamie’s Italian!

February 7th, 2017

  On Saturday 3 February Andrew Parkinson and Andrew Pearson, two of our London Marathon

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Positive Preclinical Research on the Edasalonexent (CAT-1004) Program

January 6th, 2017

  Catabasis announced the publication of preclinical data on the edasalonexent program, a potential disease-modifying

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Duchenne – How do you measure a life?

Living with Duchenne – an animation

Taking charge of Duchenne

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