Roche have announced the completion of a planned, interim analysis of the data from their trials of their myostatin inhibitor (called RG6206). The …
Vamorolone gains Promising Innovative Medicine status
We’re happy to announce that the MHRA (the medicines regulator in the UK) has given Promising Innovative Medicine (or PIM) status to Vamorolone as a …
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Patron wows audiences with moving X Factor performance
We were captivated by our Patron, Martin Bashir's beautiful show stopping performance of Nat King Cole's L-O-V-E on this weekend's The Celebrity …
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Patron Martin Bashir to feature in Celebrity X Factor
We are excited to share with you that our Patron, Martin Bashir, is featuring in the forthcoming Celebrity X Factor series in the 'OVERS' …
Patron Martin Bashir to feature in Celebrity X FactorRead More
Latest results from Vamorolone trials
A late-breaking presentation at the 24th International Annual Congress of the World Muscle Society (WMS) in Copenhagen, Denmark reported top-line data …
Cross Party Group meeting on Rare, Genetic and Undiagnosed Conditions
Last week Action Duchenne attended a meeting of the Cross Party Group on Rare, Genetic and Undiagnosed Conditions at the Scottish Parliament. The …
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Accident and Emergency Admissions
Simon and Shelley had a very productive meeting with the NHS Digital team last week who lead on data gathering and analysis. This forms a key part of …
Where’s my chair?
Action Duchenne’s Where’s my chair? campaign set out to achieve full wheelchair access for people living with Duchenne. We had a fantastic …
Information about Translarna MAA
We’re working with Muscular Dystrophy UK to make sure that emerging treatments for Duchenne get to those who need them, as quickly as possible. Since …
Wave gives suvodirsen update
Wave Life Sciences announced earlier this week that Suvodirsen has been granted fast track approval by the Federal Drug Administration (the drug …
Statement from the Board of Trustees
Action Duchenne does not routinely reply directly to comments on social media. However, given the understandable interest of the community following a …
Duchenne Standards of Care: Psychosocial
At the Action Duchenne International Conference in 2018, experts from across the world gave talks on topics around the current Standards of Care for …
