Italfarmaco Group announces positive topline data from Phase 3 trial showing beneficial effect of Givinostat in patients with Duchenne muscular …
Indoor Skydiving Experience Giveaway!
*Competition now closed* A fantastic experience worth £67.99! The winner receives the equivalent free-fall time of 3 real skydives, flight …
Edgewise Therapeutics announces positive 2-month interim results from the ARCH open label study of EDG-5506 in adults with Becker muscular dystrophy (BMD)
This article summaries the 2-month interim results shared by Edgewise Therapeutics from the ARCH study, evaluating EDG-5506 in adults with Becker …
Register for ‘Yes I can’ online and residential
'Yes I can' online workshops Every fortnight, on a Wednesday at 7pm, Ravi will be joined online by expert speakers, covering a range of important …
Impact from the first fortnight on the road
What a fortnight we've had! Cambridge, Oxford, Cardiff and Swansea - you've all been wonderful! Alex and Mehreen have loved every second of …
Nationwide researchers announce restoration of full-length Dystrophin in humans
In Duchenne muscular dystrophy, dystrophin protein is absent or partially functional due to mutations in the dystrophin gene. Multiple therapeutic …
Nationwide researchers announce restoration of full-length Dystrophin in humansRead More
Transformational transition project launches
Action Duchenne are launching an aspirational project, providing young people living with Duchenne with crucial support, training, guidance and …
Paid role with RS Components
A global company RS Components has approached us as they’d like to encourage people living with Duchenne to apply for a content role with …
Pfizer to re-start its global Phase 3 Trial of Investigational Gene Therapy for Ambulatory Patients with Duchenne Muscular Dystrophy
Pfizer has announced on 28 April 2022 they have received approvals to re-start the Phase 3 study evaluating their gene therapy for Duchenne muscular …
Science on Tour – a parent’s perspective
By Jess Breeze, Duchenne mum Our first step into the community My husband and I attended a Science on Tour session shortly after we received our …
Edgewise-funded natural history trial of Becker Muscular Dystrophy (BMD) now enrolling
Edgewise Therapeutics has announced the start of an observational trial in participants with Becker Muscular Dystrophy (‘BMD’)) as assessed by …
Edgewise-funded natural history trial of Becker Muscular Dystrophy (BMD) now enrollingRead More
WIN AN EASTER HAMPER!
With spring in the air and Easter just around the corner, we are excited to share our EGGS-tra special Easter giveaway! To win an Easter Hamper (like …
