Pfizer has announced on 28 April 2022 they have received approvals to re-start the Phase 3 study evaluating their gene therapy for Duchenne muscular …
Science on Tour – a parent’s perspective
By Jess Breeze, Duchenne mum Our first step into the community My husband and I attended a Science on Tour session shortly after we received our …
Edgewise-funded natural history trial of Becker Muscular Dystrophy (BMD) now enrolling
Edgewise Therapeutics has announced the start of an observational trial in participants with Becker Muscular Dystrophy (‘BMD’)) as assessed by …
Edgewise-funded natural history trial of Becker Muscular Dystrophy (BMD) now enrollingRead More
WIN AN EASTER HAMPER!
With spring in the air and Easter just around the corner, we are excited to share our EGGS-tra special Easter giveaway! To win an Easter Hamper (like …
Project update: Supporting families through diagnosis and impossible decisions
How we’re supporting Newly Diagnosed Duchenne families through the diagnosis and the impossible decisions they are forced to make. The last year …
Project update: Supporting families through diagnosis and impossible decisionsRead More
Annual General Meeting
On Wednesday 16 March 2022 at 17:00 we had the pleasure of welcoming the Action Duchenne Members, Trustees and team to our Annual General …
Launching Riley’s film on Rare Disease Day
That's when it hits you. Riley can't run, he'll never be able to do that.Lyndsey Kaye, Duchenne Mum At 2 years old, Riley was diagnosed with …
Join Dr. David Schonfeld Webinars
How to talk to children about Duchenne - the early years We know how hard it is for Duchenne parents, carers and family members to find the 'right' …
Joint MDUK and Action Duchenne webinar with NICE and Translarna survey 2022
This year the National Institute of Health and Care Excellence (NICE) is conducting its final appraisal of Translarna (also called ataluren). This …
Joint MDUK and Action Duchenne webinar with NICE and Translarna survey 2022Read More
End of project report summary – Clinical Trials Lectureship (Newcastle)
We are delighted to report the outcomes of our grant for a Clinical Trials Lectureship. The grant, which was supported by a consortium of seven UK …
End of project report summary – Clinical Trials Lectureship (Newcastle)Read More
Gene therapy trial shows ‘statistically significant’ improvements
Sarepta Therapeutics have announced the audited, quality-controlled data reflecting all results from Part 2 of their Study SRP-9001-102 (Study 102). …
Gene therapy trial shows ‘statistically significant’ improvementsRead More
Demelza and Action Duchenne collaborative project: transition to adulthood fact-finding
Action Duchenne aims to support our young people transitioning to adulthood by providing professional-led residential and online skills …
Demelza and Action Duchenne collaborative project: transition to adulthood fact-findingRead More
