In this, the sixth interview in the series Portrait of Duchenne, our Canadian partners La Fondation La Force talked with Steve Winder at the Action …
FDA Grants Orphan Designation to Mallinckrodt’s potential drug for Duchenne
MNK-1411 is an injection composed of a formulation of tetracosactide, which is a synthetic melanocortin receptor agonist. Melanocortin receptor …
FDA Grants Orphan Designation to Mallinckrodt’s potential drug for DuchenneRead More
Sarepta and BioMarin resolve exon skipping patent litigation
Sarepta and BioMarin have executed a license agreement that provides Sarepta Therapeutics with global exclusive rights to BioMarin’s Duchenne muscular …
Sarepta and BioMarin resolve exon skipping patent litigationRead More
Raxone is the first Duchenne drug approved through the Early Access to Medicines Scheme
We are excited to share with the Duchenne community, that Santhera Pharmaceutical’s drug Raxone, is the first Duchenne drug approved via the Early …
Raxone is the first Duchenne drug approved through the Early Access to Medicines SchemeRead More
What Raxone being given approval through the EAMS scheme means for the Duchenne community
Over a number of years we have worked with Santhera Pharmaceuticals and other Duchenne charities in relation to Santhera’s Phase III Clinical Trial …
What Raxone being given approval through the EAMS scheme means for the Duchenne communityRead More
FHA Wales staff present Three Peaks cheque to Action Duchenne
Thirteen members of staff from FHA completed the challenge in around eighteen hours, six hours ahead of the twenty-four hour deadline. They raised a …
FHA Wales staff present Three Peaks cheque to Action DuchenneRead More
The impact of Translarna on our son and our family
Article by Kathy Wedell, mother of Isaac who was 13 in April 2017 when the article was written. Kathy played a leading role in the campaign …
Action Duchenne Funding Gene Therapy to Advance Treatments for Duchenne
We are thrilled to announce our funding of “UNITE-DMD”, the first gene therapy trials for Duchenne muscular dystrophy here in …
Action Duchenne Funding Gene Therapy to Advance Treatments for DuchenneRead More
An updated detailed review of our last Scotland roundtable
On Tuesday 7 February 2017, Action Duchenne hosted a Scottish roundtable at the David Lloyd centre in Edinburgh. It provided an excellent opportunity …
An updated detailed review of our last Scotland roundtableRead More
ScOT-DMD research study update
This study is only open to boys in Scotland and although sufficient numbers of patients have been recruited in Glasgow, they are still recruiting from …
RevaraGen starts enrolment for steroid alternative clinical trial
ReveraGen BioPharma have begun enrolment in the Phase 2a clinical trial of vamoroleone in the treatment of young people living with Duchenne muscular …
RevaraGen starts enrolment for steroid alternative clinical trialRead More
Ten posts in place to support UK clinical trial capacity – Newcastle plan
In December 2015, eight patient organisations, who are working to help support clinical trial development for Duchenne muscular dystrophy, announced a …
Ten posts in place to support UK clinical trial capacity – Newcastle planRead More
