Solid Biosciences has announced that the next stage of their clinical trial of a gene therapy has started, following the dose increase announced in …
Catabasis opens new UK sites in Edasalonexent trial
Catabasis has announced that their trial of edasalonexent is now open at 2 centres in the UK. Sites in Bristol and Manchester are recruiting and two …
Catabasis opens new UK sites in Edasalonexent trialRead More
Wave announces trial results and new trial plans
Wave Life Sciences have today announced the results of their early stage phase 1 clinical trial of Suvodirsen, as well as their plans for a much …
Department for Transport Meeting
Yesterday our CEO, Shelley, was invited to the House of Commons to meet with the Secretary-under-State for the Department of Transport, Nusrat Ghani …
Match funding brings Trustee’s fundraising to over £4,000
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 …
