Dyne Therapeutics Receives FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy Dyne …
Happy Mother’s Day
Happy Mother's Day Today we celebrate all of the amazing Duchenne mummies in our community and want to let you know that we see the incredible job …
Moorside Primary school’s Holi celebrations raise money for Action Duchenne
Moorside Primary School in North Yorkshire had the amazing idea of combining their Holi celebrations with fundraising for Action Duchenne. Inspired by …
Moorside Primary school’s Holi celebrations raise money for Action DuchenneRead More
Santhera submits marketing authorisation application to the UK MHRA for Valmorolone in Duchenne Muscular Dystrophy.
Santhera submits marketing authorisation application to the UK MHRA for Valmorolone in Duchenne Muscular Dystrophy On March 2, 2023 – Santhera …
Hear about Science on Tour 2023 from our Science Officers
Our Science Officers tell you more about Science on Tour 2023 Chief Scientific Officer Dr Mehreen Arif tells you all about the content of our SOT …
Hear about Science on Tour 2023 from our Science OfficersRead More
Santhera concludes agreement with French authorities on Raxone reimbursement and plans to submit request for an Early Access Program for Vamorolone
Santhera Pharmaceuticals announces that it has secured a final reimbursement agreement with the French authorities related to Raxone® (idebenone) for …
BIND Study
UCL researchers, under supervision from Prof Francesco Muntoni, are conducting research studies to better understand how Duchenne and Becker …
Yes I Can is back for 2023!
Yes I Can is back for 2023! We’re transforming transition for the future, are you with us? Are you a young person aged 14-25 living with …
Regenexbio announces phases l/ll trial of RGX-202, a novel gene therapy candidate for Duchenne muscular dystrophy.
Regenxbio has initiated Phase I/II AFFINITY DUCHENNE™ trial of RGX-202 and the company is also enrolling newly active observational screening …
Action Duchenne Launches 2023 Science Education Programme
“KNOWLEDGE is power” is the message UK charity, Action Duchenne is sending out to families through their 2023 Science on Tour. All parents …
Action Duchenne Launches 2023 Science Education ProgrammeRead More
BREAKING NEWS
NICE publishes final guidance recommending access to Duchenne muscular dystrophy treatment Translarna NICE has published final guidance …
Sofiya joins the Action Duchenne team
A very warm welcome to Sofiya Got who has joined the Action Duchenne family as our new Science Communication Coordinator. Sofiya comes to us with …
