See the smart suit that is changing children’s lives

On Rare Disease Day, watch Eli Crossley, son of our friend and Duchenne UK co-founder Emily, as he talks about – and demonstrates – the amazing technological developments that are being trialled for children with Duchenne muscular dystrophy. The ‘smart...

Solid completes acquisition of AavantiBio

We are pleased to share that Solid Biosciences has completed its acquisition of gene therapy company AavantiBio, to create a genetic medicine company focused on neuromuscular and cardiac rare disease, with a continued, strong focus on Duchenne. At this pivotal moment...

Parents urged to read new cardiac recommendations for Duchenne

DMD Care UK’s new cardiac care recommendations have been published in the BMJ Open Heart journal. All Duchenne parents and caregivers are being urged to read the new guidance and share with their networks, as protecting the heart early is crucial to preventing...

DMD Hub launches Central Recruitment Pilot Project

The DMD Hub is launching the Central Recritment Pilot Project, aiming to create a database of families interested in take part in clinical trials. On Thursday 31st March 2022, 7-8pm, Dr Michela Guglieri (Consultant Neurologist at Newcastle University and Newcastle...

Solid data shows improved motor and lung function

We are pleased to share Solid Biosciences’ latest update on the IGNITE DMD gene therapy clinical trial. Data after two years suggests that patients’ motor function and lung function are improved compared to the declines that would be expected without...

Emergency app launched for Duchenne

An ‘In Case of Emergency’ app has been launched for Duchenne patients and their families, as part of the DMD Care UK project spearheaded by Duchenne UK and the University of Newcastle. The Duchenne Research Fund is proud to be a funder and supporter of DMD...
Five-year grant to Dr Michela Guglieri: outcome report

Five-year grant to Dr Michela Guglieri: outcome report

We are delighted to report the outcomes of our grant for a Clinical Trials Lectureship. The grant, which was supported by a consortium of seven UK charities, saw the Duchenne Research Fund joined by Action Duchenne, Alex’s Wish, Duchenne Now, Duchenne UK, Harrison’s...

DRF awards £640,000 for vital psychosocial project

The Duchenne Research Fund is delighted to announce that we are awarding a £640,000 grant to fund vital psychosocial care and research as part of Duchenne UK’s DMD Care UK programme. We are so pleased to partner with Duchenne UK on this important project. The...

DRF-funded drug shows success in clinical trial

We are pleased to share that Vamorolone, an alternative medicine to corticosteroids, has shown positive results in a Phase 2 Study. Vamorolone was developed as a treatment for Duchenne muscular dystrophy, to offer the benefits of steroids, but without some of the side...