News Archive

The Burden of Duchenne muscular dystrophy

The Burden of Duchenne muscular dystrophy

Treat NMD are pleased to announce the publication of ‘The Burden of Duchenne muscular dystrophy' international study. Significant hidden costs revealed in international study of Duchenne muscular dystrophy. In the first international study of its kind, researchers have found that there are many different costs accompanying a rare condition such as Duchenne muscular dystrophy (DMD) and that there is a considerable financial burden carried by affected families. The research, published today in the journal Neurology®, was led by Newcastle University (UK) and the Karolinska Institutet (Sweden) and carried out in collaboration with patient registries for DMD in Germany, Italy, the UK and the United States. Funded by GSK, the aim of the study was to estimate the total cost of illness and the economic burden of DMD. More...

Launch of campaign Fast Forward by MDC

Launch of campaign Fast Forward by MDC

The launch by MDC of their new campaign, Fast Forward, is aimed at ensuring that treatments for Duchenne and other muscle-wasting conditions can reach patients as quickly as possible. The launch of Fast Forward comes shortly after the European Medicines Agency (EMA) recommended conditional approval to Translarna (formerly known as ataluren), a drug designed to treat the 10 - 15% of boys and young men with Duchenne muscular dystrophy caused by a ‘nonsense mutation’. Interested people are encouraged to write to their MPs and to use social media #FastForward to spread the word.

Fundraisers needed!

We’re always on the lookout for fundraisers! DFSG is all set up with JustGiving for donations and there are lots of ideas for fundraisers – big or small – at their website www.justgiving.com

Action Duchenne Conference 2014

Action Duchenne Conference 2014

Action Duchenne will be holding their annual conference on the weekend of 7th/8th November 2014 at the Institute of Education in Central London. (University of London 20 Bedford Way, London WC1H 0AL)

DFSG supports the DMD Registry

DFSG supports the DMD Registry

The UK Duchenne Muscular Dystrophy Registry was established by Action Duchenne Ltd (formerly Parent Project UK Ltd) with a Registry Manager appointed and administered by a Curator.

Call for neonatal screening for Duchenne

Call for neonatal screening for Duchenne

With the licensing of Translarna (ataluren) the first ever drug to combat Duchenne muscular dystrophy there is growing pressure for the establishment of a neonatal screening programme for Duchenne Muscular Dystrophy.

The SKIP-NMD research project

The SKIP-NMD research project

The SKIP-NMD research project started in November 2012 with the award of an EU grant. The project brought together a consortium of researchers, clinicians and companies from across the field to develop and test a new molecular patch for exon 53 of the dystrophin gene. More...