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...

World Muscle Society awards 2023 DRF prize

The World Muscle Society has once again awarded the Duchenne Research Fund prize at the annual WMS international congress, supporting a talented researcher in the Duchenne arena. The winner at the 28th congress in Charleston, South Carolina, USA, in October 2023 was...

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...

DRF co-founder raises £15,000 in first marathon

We are so proud of our co-founder, Doron Rosenfeld, who has completed his first ever marathon in 4 hours 43 minutes. Doron said: “I always promised I would never run a marathon… I never had the desire to run one, I never believed I could. But then, following...

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...