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...

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...

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...

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...