Pfizer Doses First Patient Using Investigational Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

We are excited to share the news with the Duchenne community highlighting an important advancement in the Phase 1b, Multicenter, Open-label, Single Ascending Dose Study.Read more

Soy Products Study: Your Questions Answered

Duchenne UK recently posted a publication by Professor Steve Winder investigating a nutraceutical soy project Haelan 951. Following the publication, Duchenne UK have collated some of the questions from the Duchenne community and working with Steve Winder we have answered them.Read more


VISION-DMD is an EC funded project undertaking Phase 2 clinical studies on vamorolone, a new treatment for DMD. Vamorolone (also known as VBP15) is a dissociative steroid designed to have the efficacy of a steroid, without the severe associated side effects. The first Phase 2b study site is expected to open for recruitment in April 2018Read more

An update on soy product research funded by Duchenne UK

In August 2016 Duchenne UK commissioned Professor Steve Winder at The University of Sheffield to investigate a nutraceutical soy product called Haelan 951, as a treatment for Duchenne muscular dystrophy. Results of the study published today in PLOS Currents.Read more

SUBMIT YOUR TESTIMONY: Access to psychological support for people affected by muscle-wasting conditions

The APPG would like to hear from people with muscle-wasting conditions and relatives with positive and negative experiences of accessing psychological support and the importance of being able to access this support.Read more

Duchenne UK funds further research to understand the benefits of repurposed drugs in combination as a treatment for DMD

Duchenne UK is pleased to announce funding of £200,000 for Dr Olivier Dorchies at the University of Geneva, towards a 2-year pre-clinical study into a combination therapy for DMD.Read more

Positive results published showing the promise of using Human Artificial Chromosomes to deliver gene therapy

The newly published study at University College London, focussed on precursor cells. These precursor cells can proliferate and generate new muscle cells. The team used a viral vector to deliver 2 genes to the precursor cells.Read more

Duchenne UK receives Orphan Drug Designation for Metformin and L-Citrulline as a treatment for Duchenne Muscular Dystrophy

The European Commission have granted Orphan Drug Designation to Duchenne UK for the combination of Metformin and L-Citrulline as a treatment for DMDRead more

Lisa Kuhwald joins Duchenne UK's team as our Advocacy Support Officer

We are very pleased that Lisa Kuhwald will be joining Duchenne UK as our Advocacy Support Officer. Lisa will represent Duchenne UK at various meetings, and will provide support and advice on advocacy issues within the Duchenne community for Duchenne UK.Read more

The MHRA invite patients and carers to contribute to discussions on whether to maintain positive scientific opinion for Raxone

We need your testimony, in particular if you are receiving Santhera’s drug Raxone under EAMS, as part of the SIDEROS clinical trial or would like access Raxone for yourself or child.Read more

International Women’s Day: World's Strongest Mothers

Today, on International Women’s Day, we want to pay tribute to the World's Strongest Mums – those who we have met on our journey since our own sons were diagnosed. And those who we haven’t met but with whom we stand side by side, in our fight to end Duchenne muscular dystrophy.Read more

RCPCH Hosts an Evening of Evidence for NMDs and Launches eLearning Tool to Improve Diagnosis

The project, launched today, has developed an e-learning tool to improve diagnosis rates of NMDs. It will have particular importance for improving the age of diagnosis for Duchenne Muscular Dystrophy.Read more