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
Bone Health in Duchenne muscular dystrophy
Your views matter about bone health. There’s still time to take part in an important survey to improve bone health in Duchenne. The links below will …
ReveraGen receives FDA fast track designation for Vamorolone
ReveraGen has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for vamorolone (VBP15) for the treatment …
ReveraGen receives FDA fast track designation for VamoroloneRead More
