DMD Care UK explores next steps for patient care

On 29th February, Rare Disease Day, we were pleased to be part of the 2024 national meeting for DMD Care UK. More than 130 clinicians and 25 patient representatives are now involved in DMD Care UK, giving their time and expertise across no fewer than 13 working groups...

Vamorolone approved in the US

The US FDA (Food and Drug Administration) has approved Vamorolone for the treatment of Duchenne muscular dystrophy in children and adults aged 2 and over. It will be sold under the brand name Agamree and is expected to be available in the US in early 2024. This...

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...

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...
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...
Gavriel Rosenfeld talks to Dr Ronni Cohn about living with Duchenne

Gavriel Rosenfeld talks to Dr Ronni Cohn about living with Duchenne

On Wednesday 17th March the Duchenne Research Fund hosted a virtual evening to share updates with supporters about research funded by the charity. Our co-founders Kerry & Doron Rosenfeld welcomed hundreds of viewers from across five continents who came together...

DRF grants £40,000 to DMD Care UK project

We are delighted to announce that Duchenne Research Fund has awarded £40,000 to the newly launched DMD Care UK, partnering with the project’s founders Duchenne UK and Joining Jack. Together our three charities will invest £130,000 in the nationwide initiative...

Two-year Vamorolone study completed

Santhera Pharmaceuticals has announced that partner ReveraGen Biopharma has completed a long-term, open-label extension study of 24 months duration with Vamorolone in patients with Duchenne muscular dystrophy. Including 6 months treatment in the preceding study,...