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...

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...

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...

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...