We are doing some interviews on how Duchenne muscular dystrophy affects people and their lives, and, in particular, what aspects are most important in determining a good quality of life in people with DMD. The study will involve taking part in an interview with a researcher from the University of Sheffield.Read more
The Cheshire Dash took place in July, it is a 2-day challenge where 90 cyclist rode 200 miles across the beautiful Cheshire countryside AND raised over £112,000 for Duchenne UK. The event was organised by the Cheshire Flyers on behalf of Transforming Lives Charity. The Duchenne Dash spin off event will help us End Duchenne.Read more
A recent review reported less than 1% of published clinical trials contained data on patient engagement. We recognise the importance of patient engagement in research for DMD. After all, who really understands the burden of DMD better than patients and caregivers themselves?Read more
Alex discusses with Vision-DMD how the charities have directly funded clinical trials and research projects, co-funded clinical trial staff, and supplied direct research grants. She also discusses how DMD Hub expands trial capacity in the UK and our work to combat delays in drugs reaching the patients, instigating Project Hercules.Read more
Sarepta have been asked by the FDA to put on temporary hold their clinical trial looking at the safety and efficacy of their gene therapy treatment. The Phase 1/2a clinical trial has been put on hold due to an 'out-of-specification production lot'. We have written a summary of their press release to help the Duchenne community better understand the situation.Read more
Earlier this month, a team of 9 wonderful supporters embarked on the National 3 Peaks Challenge: climbing Ben Nevis, Scafell Pike and Snowdon within 24 hours to raise money for Project GO and Duchenne UK. Project GO have recently joined Duchenne UK as a family fund to raise awareness and vital funds to END DUCHENNE.Read more
Duchenne UK and Joining Jack have granted Professor Steve Winder a further £60,000 to continue to investigate the use of soy products as a treatment for DMD. We have now invested £135,000 towards Professor Winder’s investigation.Read more
Last week we attended a Patient Group Consultative Forum (PCGF) at the Medicines and Healthcare Products Regulatory Agency (MHRA). The meeting covered topics including the early access to medicines scheme (EAMS) and Brexit. Read our summary of the meeting.Read more
In this second blog, we focus on Care Considerations for DMD. Alex Johnson, our co-founder, took the following notes at various sessions at PPMD’s annual conference. We are working with other patient organisations to create the UK family friendly guide to understand the new and updated advice on how to care for your child.Read more
Blog 1: Gene therapy for DMD/BMD. The Duchenne community gathered in Arizona for PPMD's annual conference to learn more about the latest developments following exciting preliminary results from the Nationwide/Sarepta’s micro-dystrophin gene therapy. We wanted to share with you the notes taken during the presentations.Read more
Duchenne UK has worked closely with the team in Switzerland to help get this trial up and running, and has committed £575,000, along with E-Rare, Duchenne Parent Project in Holland, and the Monaco Association against Muscular Dystrophy.Read more
In recent years, our community has watched with hope as pre-clinical data has emerged showing the promise of gene therapy as a valuable treatment for everyone living with Duchenne Muscular Dystrophy. The year began with much hope: THREE companies, Solid Biosciences, Sarepta and Pfizer, began dosing patients in separate clinical trials using this approach. This week has seen exciting developments.Read more