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  • Challenge 79
  • About Us
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News

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Summit Presents New 24-Week Analyses from PhaseOut DMD trial

April 20, 2018 by abzali123

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

April 19, 2018 by abzali123

Capricor Therapeutics today announced that researchers found that repeat dosing of the company’s proprietary cellular therapy yields an increase …

Capricor announces repeat dosing of CAP-1002 increases exercise performance in Duchenne mouseRead More

Early data shows Eteplirsen tolerated safely in young infant with Duchenne

April 19, 2018 by abzali123

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

April 19, 2018 by abzali123

"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

April 18, 2018 by abzali123

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

April 12, 2018 by abzali123

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

April 11, 2018 by abzali123

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

April 6, 2018 by abzali123

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

April 4, 2018 by abzali123

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

April 4, 2018 by abzali123

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

April 4, 2018 by abzali123

‘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

March 23, 2018 by abzali123

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 …

Bone Health in Duchenne muscular dystrophyRead More

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