First Patients Enrolled in UK’s Early Access to Medicines Scheme for Santhera’s Raxone® in Duchenne Muscular Dystrophy (DMD)

Duchenne UK is pleased to share an update from Santhera Pharmaceuticals that the first DMD patients have been enrolled into the UK’s EAMS for Raxone®.Read more

Submit your personal stories about Ataluren/Translarna and help our friends in the USA

UK families who have participated in the Ataluren/Translarna trials and/or are currently receiving Ataluren/Translarna are asked if they would like the opportunity to submit their personal experiences to the FDA in the United States.Read more

Duchenne UK responds to the Life Sciences Industrial Strategy

The Government has today, Wednesday 30 August, published the ‘Life Sciences Industrial Strategy’. The strategy sets out how the UK can exploit and rapidly grow its life sciences industry over the next decade.Read more

Duchenne UK hosts first Patient Information Day

We invited patients and caregivers to a free information day at Alder Hey Children's Hospital, to share with them some of the trials we've funded, update them on the work we are doing, and to give advice and information about how to access research and clinical trials for DMD.Read more

Duchenne UK announces funding for a clinical trial to start this year, to study the effects of Tamoxifen in Duchenne Muscular Dystrophy

Duchenne UK is pleased to announce that it is funding a clinical trial to assess the safety and efficacy of Tamoxifen as a potential treatment for DMD.Read more

Team Felix raise £57,000 at FeFest

We would like to say a big big THANK YOU to Lisa Kuhwald and Team Felix for the success of FeFest. Lisa organised a weekend festival for 500 guests to help raise money and awareness for Duchenne UK. There was great music, food and fun with friends and family.Read more

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 Patient Engagement and Clinical Care

Duchenne UK is pleased to announce that Naomi Litchfield (nee Antcliff) is joining as Director of Patient Engagement and Clinical Care. 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