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...

FDA approves first gene therapy for Duchenne

It has been a busy time in the Duchenne arena. In the US, the FDA has granted accelerated approval for the gene therapy treatment SRP-9001. This is the first time a gene therapy for Duchenne has been approved anywhere in the world, and represents a very exciting step...

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...

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...
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...

Santhera and ReveraGen share latest vamorolone study results

We are pleased to share the announcement from Santhera Pharmaceuticals and ReveraGen BioPharma of new topline results after completion of the VISION-DMD study at week 48. VISION-DMD was a pivotal double-blind Phase 2b study designed to demonstrate efficacy and safety...