News

New information to help families from Muscular Dystrophy UK

New information to help families from Muscular Dystrophy UK

Muscular Dystrophy UK has launched the second edition Adaptations manual: for children and adults living with muscle-wasting conditions. Families regularly approach Muscular Dystrophy UK with questions about the best adaptations to get for themselves and for family members. The second edition of the adaptations manual aims to answer those questions.

TEN POSTS IN PLACE TO SUPPORT UK CLINICAL TRIAL CAPACITY

TEN POSTS IN PLACE TO SUPPORT UK CLINICAL TRIAL CAPACITY

In December 2015, eight patient organisations, who are working to help support clinical trial development for Duchenne muscular dystrophy, announced a total of £1.2million of investment, to fund a total of 16 clinical posts. Today we are announcing that TEN of those SIXTEEN posts have now been recruited. The posts are at Great Ormond Street Hospital NHS Foundation Trust in London (GOSH), The John Walton Muscular Dystrophy Research Centre at Newcastle University, University Hospitals Bristol NHS Foundation Trust and Alder Hey Children’s Hospital in Liverpool.

The Newcastle  Plan

The Newcastle Plan

Charities announce £1.2million investment in key clinical posts at leading Neuromuscular Centres for Duchenne Muscular Dystrophy. Eight patient organisations, who are working to eradicate Duchenne Muscular Dystrophy, are funding a total of 16 clinical posts at Great Ormond Street Hospital in London, The John Walton Muscular Dystrophy Research Centre at Newcastle University and Alder Hey Children’s Hospital in Liverpool, to help support clinical trial development for Duchenne Muscular Dystrophy. The DFSG are pleased to be involved with this project and although not supporting financially as this is not in our remit, we are working closely with all of the charities in a n advisory capacity .

NICE has recommended funding on the NHS in England for Translarna.

NICE has recommended funding on the NHS in England for Translarna.

NICE has recommended funding on the NHS in England for Translarna. This historic decision would make available on the NHS the first ever drug to treat an underlying genetic cause of Duchenne muscular dystrophy. Translarna would treat boys whose Duchenne is caused by a nonsense mutation, who are aged five years and over and who are still able to walk. In order to give time for NHS England and the drug company, PTC Therapeutics, to agree an final acceptable price, NICE has set July as the date where its recommendation will become final. We’ll then be pushing NHS England to implement this decision in order to get the drug to boys in clinic as quickly as possible.

SKIP-NMD – a new Duchenne clinical trial starting

SKIP-NMD – a new Duchenne clinical trial starting

A new clinical trial to test a molecular patch skipping exon 53 in boys with Duchenne Muscular Dystrophy is starting. The molecular patch could potentially be used to restore dystrophin production in about eight percent of boys with Duchenne muscular dystrophy.

News Archive

News Archive

Archive of past news items and events. News items are moved here when they have passed but still have content that will be of use to and of interest to our readers.