Sarepta Therapeutics, Inc. today provided an update on the regulatory progress of ELEVIDYS (delandistrogene moxeparvovec-rokl), its gene therapy for …
Start your Summer as an AD Champion at the Parellel Windsor Festival of Inclusivity
Start your Summer as an Action Duchenne Champion Action Duchenne are proud to be a Parallel charity partner for their flagship Festival of …
Start your Summer as an AD Champion at the Parellel Windsor Festival of InclusivityRead More
SOT OXFORD
SOT OXFORD Book your FREE place on our Oxford Science workshop on 11th June, running from 10.30am- 3:00pm. …
Come together with the Duchenne community at Action Duchenne’s Annual International Conference 2024
Come together with the Duchenne community at Action Duchenne's Annual International Conference 2024 Friday 8th & Saturday 9th November 2024, …
Dyne Therapeutics Announces New Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD
Dyne Therapeutics announced on the 20th of May, positive clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic …
Calling everyone from Scotland and Wales – Tell us what YOU think!
Tell us what YOU think We are contacting all of our families, clinicians, schools and care professionals across Scotland and Wales as we try to …
Calling everyone from Scotland and Wales – Tell us what YOU think!Read More
MHRA Translarna Survey
Introduction In 2014, the European Medicines Agency (EMA), granted a ‘Conditional Marketing Authorisation’ to Translarna for the treatment of …
PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation
PTC Therapeutics has announced today that the European Commission (EC) has decided not to adopt the European Medicine Agency (EMA) Committee for …
Spring Webinar : BIND Study
The BIND project: Learning and behaviour challenges in Duchenne and Becker Muscular Dystrophies Join us on Wednesday 29th May at 5:30 pm for the …
Links and Resources
Here are some links to other charities and organisations who have been part of Yes I Can sessions, or that you might find useful. Pathfinders …
Science on Tour 2024 – Register your Interest
Behind the scenes at Action Duchenne, our Science Communication team have been working hard to put together 2024 Science on Tour Programme. The …
Spring Webinar – Dr Vasantha Gowda ‘DMD and Emergencies’
Join us on Saturday 11th May at 5pm for the next in our series of webinars. Dr Vasantha Gowda, consultant in paediatric neurology and neuromuscular …
Spring Webinar – Dr Vasantha Gowda ‘DMD and Emergencies’Read More
