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 …
Santhera and ReveraGen Complete NDA Submission to FDA for Vamorolone in Duchenne Muscular Dystrophy
Ad hoc announcement pursuant to Art. 53 LR Pratteln, Switzerland, and Rockville, MD, USA, October 27, 2022 – Santhera Pharmaceuticals (SIX: …
Important update for DMD parents and carers
Important update for DMD parents and carers:DMD Care UK’s new recommendations for cardiac care have been published in the BMJ Open Heart today.You can …
Peer-to-peer support – transitioning to secondary
This week's Friday Hive support session (as part of the All-through Support project) we talked about transitioning to secondary school and how to make …
NICE publishes draft guidance on access to Duchenne treatment Translarna
NICE has published draft guidance stating that although Translarna (also known as ataluren) is clinically effective and an innovative treatment, there …
NICE publishes draft guidance on access to Duchenne treatment TranslarnaRead More
Questions at the Conference
Action Duchenne is providing a platform for you, the Duchenne community, to ask questions from the leading Pharmaceutical companies driving …
Newly diagnosed family online event
Building your foundation of Duchenne knowledge Online via ZoomSaturday 22nd October 202214:00 - 17:00 GMTAll welcome More information An …
