We are delighted to announce that the MHRA have decided to maintain a positive opinion on the Early Access to Medicines Scheme for Raxone to treat …
Thank You – An announcement on the Early Access to Medicines Scheme for RaxoneRead More
Summit Presents New 24-Week Analyses from PhaseOut DMD trial
Data show correlation between decrease in muscle damage and reduction in muscle inflammation in patients treated with ezutromid Summit Therapeutics …
Summit Presents New 24-Week Analyses from PhaseOut DMD trialRead More
Capricor announces repeat dosing of CAP-1002 increases exercise performance in Duchenne mouse
Capricor Therapeutics today announced that researchers found that repeat dosing of the company’s proprietary cellular therapy yields an increase …
Early data shows Eteplirsen tolerated safely in young infant with Duchenne
Muscular Dystrophy News Today report in their article that 'Exondys 51' ('Eteplirsen' in the UK), was according to preliminary data, safe and well …
Early data shows Eteplirsen tolerated safely in young infant with DuchenneRead More
Summit Completes Dosing of Ezutromid in PhaseOut DMD Clinical Trial
"We believe the early improvements seen in muscle health in the interim data from PhaseOut DMD indicate ezutromid is reducing DMD disease severity. In …
Summit Completes Dosing of Ezutromid in PhaseOut DMD Clinical TrialRead More
Solid address Duchenne community with update on IGNITE DMD Clinical Trial
Solid Biosciences have written to the Duchenne community to provide an update on their IGNITE DMD clinical trial. Below is a transcript of this letter …
Solid address Duchenne community with update on IGNITE DMD Clinical TrialRead More
Pfizer doses first patient using investigational mini-dystrophin gene therapy
Pfizer Inc. has initiated a Phase 1b clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in boys with Duchenne muscular …
Pfizer doses first patient using investigational mini-dystrophin gene therapyRead More
Wave enter into collaboration with Deep Genomics to expand research programme
This new collaboration builds on Wave’s ongoing research and development in splice correction programmes, including its lead DMD programme, …
Wave enter into collaboration with Deep Genomics to expand research programmeRead More
VISION-DMD newsletter launched at Phase 2 of clinical trial of Vamorolone
Action Duchenne supported the preclinical funding and Phase 1 clinical trials of this innovative steroid-like intervention on Duchenne muscular …
VISION-DMD newsletter launched at Phase 2 of clinical trial of VamoroloneRead More
Adelphi Values would like to interview people from the Duchenne community
Action Duchenne have been asked by Adelphi Values, to help recruit people from the Duchenne community, for their important study. The UK has a …
Adelphi Values would like to interview people from the Duchenne communityRead More
Give your valuable experiences of caring and supporting a young person with Duchenne
The Ulster University have approached Action Duchenne and our wonderful community to take part in an important research study which could benefit the …
Give your valuable experiences of caring and supporting a young person with DuchenneRead More
‘Together for short lives’ launches new transition checklist
‘A checklist to a good transition’ joins the suite of resources compiled by 'Together for Short Lives', aimed at improving the experiences …
‘Together for short lives’ launches new transition checklistRead More
