Entrada Therapeutics, a company developing treatments for rare diseases, reports encouraging preliminary data from a Phase 1 clinical trial …
Action Duchenne’s Science on Tour Team visit Ashford School
Action Duchenne Science on Tour Team visit Ashford SchoolA new addition to this years’ Science on Tour is our school visits, part of our ongoing …
Action Duchenne’s Science on Tour Team visit Ashford SchoolRead More
Make a Difference – Join Our Charity Running Events!
Are your employees ready to lace up their running shoes and support a great cause? 🌟 We invite your team to join us at one of our upcoming charity …
Make a Difference – Join Our Charity Running Events!Read More
Do you live in Scotland or Wales? Join our Focus Groups to tell us what support YOU need!
Do you live in Scotland or Wales? Join our Focus Groups to tell us what support YOU need!Improving the lives of everyone that lives with DMD is at the …
Do you live in Scotland or Wales? Join our Focus Groups to tell us what support YOU need!Read More
Sarepta Therapeutics Announces Positive Update on ELEVIDYS (delandistrogene moxeparvovec-rokl) Regulatory Progress for Duchenne Muscular Dystrophy (DMD)
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 OXFORDBook 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 2024Friday 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 thinkWe are contacting all of our families, clinicians, schools and care professionals across Scotland and Wales as we try to gather …
Calling everyone from Scotland and Wales – Tell us what YOU think!Read More
MHRA Translarna Survey
IntroductionIn 2014, the European Medicines Agency (EMA), granted a ‘Conditional Marketing Authorisation’ to Translarna for the treatment of patients …
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 …
