We are thrilled to announce our funding of “UNITE-DMD”, the first gene therapy trials for Duchenne muscular dystrophy here in …
Action Duchenne Funding Gene Therapy to Advance Treatments for DuchenneRead More
An updated detailed review of our last Scotland roundtable
On Tuesday 7 February 2017, Action Duchenne hosted a Scottish roundtable at the David Lloyd centre in Edinburgh. It provided an excellent opportunity …
An updated detailed review of our last Scotland roundtableRead More
ScOT-DMD research study update
This study is only open to boys in Scotland and although sufficient numbers of patients have been recruited in Glasgow, they are still recruiting from …
RevaraGen starts enrolment for steroid alternative clinical trial
ReveraGen BioPharma have begun enrolment in the Phase 2a clinical trial of vamoroleone in the treatment of young people living with Duchenne muscular …
RevaraGen starts enrolment for steroid alternative clinical trialRead More
Ten posts in place to support UK clinical trial capacity – Newcastle plan
In December 2015, eight patient organisations, who are working to help support clinical trial development for Duchenne muscular dystrophy, announced a …
Ten posts in place to support UK clinical trial capacity – Newcastle planRead More
PM replies to parents and patients on Translarna
After submitting a letter to No.10 Downing St on June 9, the Prime Minister David Cameron sent a host of replies to Action Duchenne yesterday to pass …
Access to Medicines and the Campaign for Translarna (Ataluren) timeline
Since the start of 2015, Action Duchenne campaigned tirelessly for Translarna (ataluren) to be made available, exerting maximum external pressure upon …
Access to Medicines and the Campaign for Translarna (Ataluren) timelineRead More
Action Duchenne working with Scottish Family to get Translarna approved by SMC
Ross Munro, living with Duchenne, was first enrolled on the PTC Therapeutics trial of Translarna in 2008, and has been in receipt of the …
Action Duchenne working with Scottish Family to get Translarna approved by SMCRead More
The fight for Translarna goes on – Action Duchenne in parliamentary session
In light of NHS England’s non-decision on Translarna last week, Action Duchenne shared a platform with the MPS Society in parliament yesterday …
The fight for Translarna goes on – Action Duchenne in parliamentary sessionRead More
Press release: First ever treatment for rare muscle wasting condition, Duchenne Muscular Dystrophy, given conditional approval by the European Commission
Children living with a genetic muscle wasting condition called Duchenne Muscular Dystrophy (DMD) have hope of a different future after the first ever …
