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 larger phase 2/3 clinical trial of the potential drug. Read more

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 MP, to discuss the importance of the government's Inclusive Transport Strategy of July 2018 and the new Access For All programme. Read more

Match funding brings Trustee's fundraising to over £4,000

A fortnight ago, Sarah Colvin faced windy hill climbs, rain, and even two punctures, in her cycle from London to Penzance. She exceeded £2,000 in her fundraising and has now doubled her impact through company match-funding! Read more

Santhera announces plans to apply for conditional approval for Puldysa

Santhera Pharmaceuticals plans to submit an application for Conditional Approval for Puldysa (also known as idebenone and Raxone) in people living with Duchenne Read more

Sarepta Therapeutics announces preliminary results in casimersen (exon 45 skipping) trial

In a press release, Sarepta Therapeutics announced results of an interim analysis of the casimersen part of their late stage, phase 3 ESSENCE trial. Read 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 steroid regimes. Read more

Jonny Gould becomes an Ambassador for Action Duchenne

Jonny is a presenter and pundit who has appeared regularly on Sky News, ITV Sport, Talk Sport and many other media outlets over more than 30 years and has a cousin living with Duchenne Muscular Dystrophy. Read more

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! Read more

Santhera announces idebenone study results

Santhera announces long-term effectiveness of idebenone in slowing the decline of breathing function in Duchenne Read more

Raxone: A Guide for the Community

In partnership with fellow organisations, Muscular Dystrophy UK, Duchenne UK, the Duchenne Family Support Group, and DMD Pathfinders, we have produced two guides for families and healthcare professionals to educate and answer questions on the drug and EAMS process. Read more

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 Conference. Read more

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 in Cyprus, with space for 8 people to enjoy the pool, sun and gorgeous relaxing surroundings. Read more

Solid Biosciences announce preliminary trial results

Solid Biosciences have announced preliminary data from their IGNITE-DMD clinical trial that is testing the safety and effectiveness of an adeno-associated virus (AAV) gene therapy. Read more

Family urge PTC to fight for Translarna approval

Cormac Fegan, age 8, and his family were invited to give a Q&A session to mark the start of the second day of the event to over 150 attendees from all areas of the company covering all geographic areas. Read more

Call to action - Powerchair Football in Scotland

Please join the Cross Party Debate discussing Powerchair football in Scotland on Wednesday the 6th of February 2019 at 5:15pm in Scottish Parliament. Read more

HOPE-2 update from Capricor Therapeutics

Capricor Therapeutics update on it’s HOPE-2 clinical trial of CAP-1002, a potential treatment for Duchenne muscular dystrophy. Read more

What your money buys

If you're supporting one of our many Duchenne research projects, here's an idea of where your money goes, and how every penny counts. Read more

Edasalonexent update from catabasis

Catabasis has announced that the phase 3 trial of edasalonexent is now recruiting at 9 centres across the USA and that regulatory approval for the trial to start in Europe has been received. Read more

Sarepta completes accelerated approval application for golodirsen

Sarepta Therapeutics has completed the submission of a request for accelerated approval for golodirsen in the USA Read more

UNITE-DMD project update - Year 1

Update on the progress of the UNITE-DMD programme that we fund jointly with MDUK and AFM-Telefon. Read more

Wave Life Sciences announce phase 1 trial results

Wave Life Sciences has announced results of the phase 1 trial of the WVE-210201 – a potential drug that induces skipping of exon 51 of the dystrophin gene Read more

ENMC Workshop in Amsterdam

Our Director of Research, Neil attended the 238th ENMC workshop. Read more

Action Duchenne present parent's perspective to healthcare professionals

We as a charity relish every opportunity to advocate life with Duchenne to professionals in the Healthcare field. Read more