Entrada Therapeutics has shared several key milestones regarding their ELEVATE clinical trial programs. These studies are designed to evaluate the …
Entrada Therapeutics: 2026 Clinical Trial Plan & ProgressRead More
Sarepta Announces Topline Three-Year EMBARK Results: Long-Term Performance of ELEVIDYS Gene Therapy
Sarepta Therapeutics has released updated findings from its Phase 3 EMBARK study, providing a three-year assessment of the gene therapy ELEVIDYS …
Training Plans and Injury Prevention
If you have signed up for an event and registered on the official race console, you will receive email communications from the event. This will …
NICE Givinostat Appraisal Paused
The Scottish Medicines Consortium (SMC) recently announcement of their approval of Givinostat for the treatment of Duchenne muscular dystrophy (DMD) …
Bone Health and Management Guidance Update
Dr Jarod Wong from the University of Glasgow together with a group of clinical experts from the UK, with support from Action Duchenne and Muscular …
Dyne Therapeutics Announces Positive Top-Line Results from DELIVER Trial
There is encouraging news for the Duchenne community as Dyne Therapeutics has announced positive top-line results from their ongoing DELIVER clinical …
Dyne Therapeutics Announces Positive Top-Line Results from DELIVER TrialRead More
Givinostat Accepted for Restricted Use Within NHS Scotland for the Treatment of Duchenne in Patients 6 Years and Older
A significant milestone for the Duchenne community in Scotland as a new treatment option due to become available on the NHS. We are delighted to …
Introducing Action Duchenne’s new CEO: Katie Endacott
We are delighted to introduce our new CEO, Katie Endacott and to share a statement from Vicky Pleydell, Chair of Trustees: "I am delighted to …
Introducing Action Duchenne’s new CEO: Katie EndacottRead More
Action Duchenne comment on statement from NorthStar Network UK about Givinostat
Action Duchenne comment on statement from NorthStar Network UK about Givinostat Firstly, and most importantly, our thoughts are with the families of …
Action Duchenne comment on statement from NorthStar Network UK about GivinostatRead More
Community Accessibility and Support
Community Accessibility and Support Our Community Accessibility and Support stream will run on Friday 14th November. It's designed to equip and …
DMD Care UK Nutrition Working Group – Online Focus Groups
This month there will be an opportunity for everyone interested in learning more about nutrition and Duchenne to participate in two online focus group …
DMD Care UK Nutrition Working Group – Online Focus GroupsRead More
Impact of NRS Healthcare Closure: Statement from the Chair of Action Duchenne, Vicky Pleydell
Urgent action needed to protect families affected by Duchenne muscular dystrophy I am deeply concerned about the devastating impact the collapse of …
