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...
The Duchenne Research Fund was pleased to participate in Duchenne UK’s latest parent information day, held in March in central London. Dozens of families attended from all over the UK, and one even made the trip from Boston to hear about the latest research and...
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...
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...
We are delighted that the ground-breaking KineDMD activity monitoring study we funded has been published in the prestigious scientific journal Nature Medicine. As the publication succinctly states: “Artificial intelligence has the potential to revolutionise...
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 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...
Solid Biosciences has announced its entry into a definitive merger agreement to acquire AavantiBio, to create a genetic medicines company focused on neuromuscular and cardiac rare diseases, maintaining a strong focus on Duchenne. In a letter to the Duchenne community,...
We are delighted to share that Great Ormond Street Hospital Trust has agreed to make permanent a physiotherapist role that the Duchenne Research Fund has funded for the past few years. Senior physiotherapist Nicola Burnett (pictured) has been working with young people...
Dedicated specialist support for Duchenne muscular dystrophy (DMD) patients and families’ mental health and wellbeing will soon be more readily available, as part of the DMD Care UK project. DMD Care UK is a nationwide initiative aiming to ensure that every person...
