A fortnight ago, one of our wonderful Trustees, Sarah Colvin, braved the elements and completed her cycle from London to Penzance. Facing windy hill …
Match funding brings Trustee’s fundraising to over £4,000Read More
Santhera announces plans to apply for conditional approval for Puldysa
Santhera Pharmaceuticals has announced plans to submit an application for Conditional Approval for Puldysa (also known as idebenone and Raxone) for …
Santhera announces plans to apply for conditional approval for PuldysaRead More
Sarepta Therapeutics announces preliminary results in casimersen (exon 45 skipping) trial
In a press release earlier, Sarepta Therapeutics announced results of an interim analysis of the casimersen part of their late stage, phase 3 ESSENCE …
Sarepta Therapeutics announces preliminary results in casimersen (exon 45 skipping) trialRead More
Study investigates bone health in Duchenne
A recently published study has found that boys treated with daily Deflazacort (a corticosteroid) have more fractures than boys treated with other …
Jonny Gould becomes an Ambassador for Action Duchenne
We are delighted to announce that television and radio broadcaster Jonny Gould has become an ambassador for Action Duchenne. Jonny is a presenter and …
Jonny Gould becomes an Ambassador for Action DuchenneRead More
Annemeike standards of care video news article
Trevor and Lucy become Ambassadors for Action Duchenne
We are delighted to announce our friends and long-time supporters of the charity, Trevor and Lucy, are our new Action Duchenne Ambassadors! Lucy …
Trevor and Lucy become Ambassadors for Action DuchenneRead More
Raxone: A Guide for the Community
Raxone (also known as Idebenone) is a drug produced by Santhera Pharmaceuticals, which treats several conditions and has the potential to treat …
Santhera announces idebenone study results
In a press release, Santhera Pharmaceuticals has announced results from the SYROS study, where long-term treatment with idebenone (also known as …
Duchenne Parent Project International Conference
This weekend our Director of Research, Neil Bennett, and our CEO, Shelley Simmonds, are in Rome attending the Duchenne Parent Project International …
Golf Club raise £3,050 in villa raffle
Last weekend, our wonderful supporters Stoke By Nayland Golf Club held their much anticipated raffle draw. The prize was a week in a luxury villa …
Solid Biosciences announce preliminary trial results
Solid Biosciences have announced preliminary data from their phase 1/2 IGNITE-DMD clinical trial that is testing the safety and effectiveness of …
Solid Biosciences announce preliminary trial resultsRead More
