Santhera’s Raxone® Receives First Positive EAMS Scientific Opinion from UK’s MHRA in Duchenne Muscular Dystrophy

Duchenne UK is pleased to share news from Santhera Pharmaceuticals regarding the progress of Raxone. MHRA has granted Raxone a positive scientific opinion through the EAMS.Read more

Duchenne UK launches the INNOVATE grant call, worth up to £1million

Duchenne UK is pleased to announce the launch of its ‘Innovate’ Grant Call, and invites proposals for funding projects up to £1million, dedicated to finding new treatments for Duchenne muscular dystrophy (DMD).Read more

Santhera Provides Update on Timeline for Application of Raxone® in Duchenne Muscular Dystrophy in Europe

Santhera announces an updated timeline for the ongoing assessment by the Committee for Medicinal Products for Human Use of its application for Raxone®Read more

Duchenne UK welcomes new Director of Research and Patient Engagement

Duchenne UK is pleased to announce that Naomi Litchfield (nee Antcliff) is joining as Director of Research and Patient Engagement. Naomi will be known to families who have taken part in research at Great Ormond Street Hospital.Read more

New Preclinical Data Support SGT-001 As A Novel Treatment Approach For Duchenne Muscular Dystrophy

Duchenne UK is delighted to share this announcement from Solidbio about their investigational micro-dystrophin gene therapy, which is planned to go into clinical trials later this year.Read more

Patient centered outcome measurements in rare disease trials: Challenges and potential solutions

Our co-founder Alex Johnson spoke about patient perspective on outcome measures at the International Clinical Trials Methodology Conference. This article contains the abstract.Read more

Duchenne UK launches patient survey

As part of our community engagement work Duchenne UK launches patient survey to better understand the hopes and expectations of patients and caregivers who are considering taking part in clinical trials. This will be the first survey in our series.Read more

Duchenne UK finance innovative research project to explore elastase inhibitors as a potential treatment for DMD

Duchenne UK is co-funding a study into the use of elastase inhibitors to treat the symptoms of Duchenne Muscular Dystrophy (DMD) in partnership with Joining Jack and Charley's Fund.Read more

Duchenne UK is pleased to be supporting the upcoming TACT meeting

TACT is a unique multi-disciplinary international group of academic, industry drug development experts and representatives of patient foundations and institutional governmental scientific research centers.Read more

Duchenne UK is investing £543,314 to fund 5 posts at the John Walton Muscular Dystrophy Research Centre and Royal Victoria Infirmary in Newcastle

Alex and Emily went to Newcastle to see the newly refurbished Clinical Research Facility.Read more


Duchenne UK is hosting its first Duchenne Information Day in the North West. We’re hosting it in collaboration with Alder Hey Children's Hospital, the National Institute for Health Research Alder Hey Clinical Research Facility, the DMD Hub and TREAT-NMD.Read more

Duchenne UK announces new digital innovation funding as part of commitment to supporting clinical trial development

Duchenne UK will fund the development of digital technology to develop and evaluate new approaches to measuring efficacy and safety in clinical trialsRead more