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 …
Santhera and ReveraGen Announce FDA Acceptance of New Drug Application for Vamorolone in Duchenne Muscular Dystrophy
Santhera Pharmaceuticals and ReveraGen BioPharma, Inc announce that the U.S. Food and Drug Administration (FDA) has accepted the new drug application …
End of Life and Bereavement Support Survey
Parents and family members have told us once their loved ones have passed on they no longer feel part of the Duchenne community as they were …
Roche DMD Team: End of Year Duchenne Community Update
As we approach the end of the year, Roche are providing an update to the Duchenne Community about their activities surrounding delandistrogene …
Roche DMD Team: End of Year Duchenne Community UpdateRead More
Sarepta Theraputics Gene Therapy Granted Priority Review by FDA
Sarepta Therapeutics announced that the U.S. FDA has accepted the Company's Biologics License Application for SRP-9001 gene therapy for …
Sarepta Theraputics Gene Therapy Granted Priority Review by FDARead More
Support our new project this Christmas
Recently my son’s needs changed, his school was struggling and without the correct equipment and training in place a decision by the local authority …
