Sarepta Therapeutics has announced the latest results from their gene therapy trial

Sarepta therapeutics has announced a further set of preliminary results from their early-stage gene therapy trialRead more

Catabasis starts Phase 3 trial of edasalonexent

Catabasis announce that a Phase 3 trial of edasalonexent has started, with trial centres to open shortly – first in the USA and then in Europe.Read more

Sarepta Therapeutics gene therapy trial to continue

The FDA has lifted the Clinical Hold that was issued to Sarepta Therapeutics phase I/II gene therapy trialRead more

Sarepta receives negative opinion following CHMP review of eteplirsen

Sarepta has received a negative opinion of eteplirsen following a second review by the European Medicine's Agency Committee for Medicinal Products for Human Use (CHMP).Read more

Vamorolone trial results published

The results of the first clinical trial of vamorolone (which finished in 2016) in people living with Duchenne were published last weekRead more

Berardelli family fundraise at Lochaber Agricultural Show

Elspeth and Paolo have been dedicated fundraisers for many years now following their son Georgio's diagnosis with Duchenne.Read more

Gene editing restores dystrophin production in dog model of Duchenne

Researchers from the United States and London’s Royal Veterinary College have demonstrated that gene editing techniques can restore dystrophin production in a dog model of Duchenne.Read more

World Duchenne Awareness Day 2018

Tomorrow, 7th September, marks annual Duchenne Awareness Day – a day in which the entire community comes together with one clear mission – for our families and raising the profile of Duchenne muscular dystrophy to a wider audience.Read more

Pfizer to discontinue trials of domagruzumab in Duchenne

Pfizer has announced that trials of domagrozumab are to be discontinued after it failed to show effectiveness in a phase 2 clinical trial.Read more

Wave gain Orphan Drug status for WVE-210201

Wave Life Sciences has announced that the Drug Regulator in the USA – the FDA - has granted both orphan drug designation and rare paediatric disease designation for WVE-210201 – a potential exon skipping drug.Read more

Take part in 5 minute online corticosteroids study

Please take a few minutes to complete this short, anonymous online survey to find out about the side effects of steroids that impacts most on quality of life in people living with Duchenne.Read more

PTC use ataluren trial data to investigate steroids

PTC Therapeutics publishes a paper that uses data from the ataluren trial to investigate the effectiveness of deflazacort and prednisolone.Read more

Clinial hold in Sarepta gene therapy trial

Sarepta Therapeutics has announced that the FDA has issued a Clinical Hold letter for the company’s phase I/II gene therapy trial.Read more


We have been offered a number of paid internships for young people living with Duchenne. The positions are available this summer across a range of departments within a well-known organisation.Read more

EAMS, Raxone and the MHRA

Last week our Director of Campaigns & Strategy, Peter Duffy, attended a meeting at The Medicines and Healthcare products Regulatory Agency (MHRA), as part of the Patient Group Consultative Forum.Read more

Catabasis announces plans for global trial of edasalonexent

Catabasis announces plans to start a global phase 3 trial to test the safety and effectiveness of a potential drug called edasalonexent in people living with Duchenne muscular dystrophy.Read more

Action Duchenne skydive 2018 was amazing

A huge THANK YOU to the 12 amazing fundraisers who jumped out of planes across the country for Duchenne muscular dystrophy this weekend!Read more

Cambridgeshire Dad pushes himself to the limit

A huge thank you and well done to the incredible Alexis Ellison who completed his 24 hour mammoth bike ride - cycling the 9 mile road back and forth from his home town of St Neots to Godmanchester for a whole 24 hours, completely unsupported!Read more

Summit announces results of PhaseOut DMD clinical trial of Ezutromid

Although Ezutromid was safe and well tolerated, after 48 weeks the trial clearly showed that there was no clinical benefit for those who received Ezutromid. As a result, Summit have decided to discontinue the development of Ezutromid.Read more

ReveraGen BioPharma announces preliminary results of Vamorolone trial

ReveraGen BioPharma has announced encouraging preliminary results of their early stage clinical trial of Vamorolone, a potential drug that researchers hope could offer similar benefits to steroids with reduced side effects.Read more


We are dedicated in delivering our vision to you: to have a world where lives are no longer limited by Duchenne or Becker Muscular Dystrophy. Developing effective treatments for all, building a supportive community, and striving for a more inclusive society will always be at the heart of what we do.  Join us in raising the profile of Duchenne, funding pioneering research and clinical trials and educating society on inclusion and disability rights.Read more

Santhera announces renewal of Raxone EAMS

Santhera has announced that the MHRA (the UK’s drug and medicines regulator) has granted a renewal of the Early Access to Medicines Scheme scientific opinion for Raxone.Read more

Sarepta announce early, preliminary gene therapy trial results

Sarepta therapeutics has announced preliminary results of a gene therapy trial that is using a harmless virus to deliver a small but functional dystrophin gene (micro-dystrophin) to the leg muscles of people living with Duchenne. The early results are encouraging, but it is too early to say whether the potential treatment will prove to be safe or effective.Read more

CEO attends EURORDIS Summer School

Last week our incoming CEO, Shelley Simmonds, completed a 5 day intensive training programme in Barcelona, having been selected to take part in the 'Expert Patient and Researcher EURORDIS Summer School'.Read more