Harry Hill, one of our amazing Patrons, sent us a video which we proudly played at the Gala Dinner on Friday at the Action Duchenne International …
An unforgettable weekend at #ADCON19
Thank you to each and every person who attended the Action Duchenne International Conference 2019. We have all had such a wonderful weekend with …
Solid gene therapy trial placed on hold
Solid BioSciences has announced that the IGNITE DMD trial - a Phase I/II clinical trial of the company’s SGT-001 gene therapy - has been placed …
Thank you for helping us build a community
Wow, we've had a busy few weeks here at AD HQ, delivering one of our 3 key objectives of 'building a community'. Our team have barely touched the …
Roche halts development of myostatin inhibitor
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 …
Patron wows audiences with moving X Factor performanceRead More
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 …
Cross Party Group meeting on Rare, Genetic and Undiagnosed ConditionsRead More
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 …
