Sarepta Therapeutics Announcement: Positive Preliminary Results from the First Three Children Dosed in the Phase 1/2a Gene Therapy Micro-dystrophin Trial

Sarepta announced that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from its Phase 1/2a gene therapy clinical trialRead more

Jack Wills supports Duchenne UK

We are delighted to announce our charity partnership with British fashion and lifestyle brand, Jack Wills. To launch the partnership, Jack Wills is hosting their own in-store Duchenne Dash TOMORROW, Friday 1st of June. 10 Jack Wills stores and two Head Office teams will compete against each other to cycle 300km (the distance to Paris) on stationary bikes.Read more

Duchenne UK announces £46,930 to fund the expansion of clinical trial capacity at Birmingham Heartlands Hospital

We are delighted that Birmingham Heartlands Hospital is joining the DMD Hub. We will fund consultant time which will be dedicated to supporting clinical trials, as well as funding physiotherapy equipment for clinical trials.Read more

Duchenne UK attends the opening of new Clinical Research Facility at Leeds Children's Hospital and announces £108,324 of funding for research posts

Last Thursday, our co-founder Alex Johnson attended the opening of the Clinical Research Facility at Leeds Children's Hospital which has recently joined the DMD Hub.Read more


Duchenne UK is pleased to announce the launch of the DMD Hub website,, the go-to website for clinical trial information for DMD). The mission of the Hub is to ensure that all patients with DMD, including children and adults, have access to clinical research opportunities.Read more

Delay in European Access to Sarepta's Exondys 51

Sarepta Therapeutics has received a negative trend vote following its CHMP oral explanation from the European Medicine Agency. The company will request a re-examination and Scientific Advisory Group to be convened. This means that efforts to bring eteplirsen to people living with Duchenne in Europe will be delayed.Read more

MHRA maintain a positive opinion on the Early Access to Medicines Scheme for Raxone

We are delighted to announce that the MHRA have decided to maintain a positive opinion on the Early Access to Medicines Scheme for Raxone to treat people living with Duchenne muscular dystrophy. Raxone remains on course to have its EAMS award renewed.Read more

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