Come together with the Duchenne community at Action Duchenne's Annual International Conference 2024Friday 8th & Saturday 9th November 2024, …
Project that aimed to combine stem cell and CRISPR technology is a success
We’re delighted to report the successful completion of our research project in Dr Yung-Yao Lin’s laboratory at Queen Mary University of London. The …
Project that aimed to combine stem cell and CRISPR technology is a successRead More
Golodirsen gains conditional approval in US
Sarepta Therapeutics has announced that the FDA (the medicines regular in the US) has granted accelerated approval to golodirsen. Golodirsen, or …
Update on UNITE-DMD project
The UNITE-DMD project, which we are co-funding alongside Muscular Dystrophy UK and AFM-Téléthon brings together several strands of research to …
Duchenne Science events back on the road in 2020
We've welcomed over 100 people to our Science on Tour events throughout 2019, these empowering and invaluable sessions have been a huge success and we …
Thank you for helping us build a community
Wow, we've had a busy few weeks here at AD HQ, delivering one of our 3 key objectives of 'building a community'. Our team have barely touched the …
UNITE-DMD project update – Year 1
Gene therapy for Duchenne muscular dystrophy aims to compensate for the lack of dystrophin by transferring a working version of the dystrophin gene …
Summit announces results of PhaseOut DMD clinical trial of Ezutromid
Earlier today Summit Therapeutics announced the results of the PhaseOut DMD clinical trial of Ezutromid. Although Ezutromid was safe and well …
Summit announces results of PhaseOut DMD clinical trial of EzutromidRead More
Take part in ventilator study and earn £75
SuAzio are currently conducting a study and are looking for individuals who live with Duchenne, or their caregivers to be part of this study. …
Give your valuable experiences of caring and supporting a young person with Duchenne
The Ulster University have approached Action Duchenne and our wonderful community to take part in an important research study which could benefit the …
Give your valuable experiences of caring and supporting a young person with DuchenneRead More
Revised Standards of Care for Duchenne muscular dystrophy
We are delighted to announce that the revised Standards of Care for Duchenne were published in The Lancet Neurology this week; a high-impact …
Revised Standards of Care for Duchenne muscular dystrophyRead More
Action Duchenne launches new Research Strategy
Action Duchenne, a leading UK-wide patient and parent-led organisation for Duchenne Muscular Dystrophy, is delighted to announce the publication of a …
