Here are some links to other charities and organisations who have been part of Yes I Can sessions, or that you might find useful. Pathfinders …
Science on Tour 2024 – Register your Interest
Behind the scenes at Action Duchenne, our Science Communication team have been working hard to put together 2024 Science on Tour Programme. The …
Spring Webinar – Dr Vasantha Gowda ‘DMD and Emergencies’
Join us on Saturday 11th May at 5pm for the next in our series of webinars. Dr Vasantha Gowda, consultant in paediatric neurology and neuromuscular …
Spring Webinar – Dr Vasantha Gowda ‘DMD and Emergencies’Read More
Annemeike Aartsma-Rus Webinar Recording
For anyone who missed the brilliant webinar hosted by Professor Annemeike Aartsma-Rus from 13th April 2024, this is the recording of her session. She …
Take part in a trial programme about behavioural and emotional difficulties
Andria Papageorgiou, postgraduate research student at The University of Surrey, is inviting you to take part in a 6 week programme for parents/carers …
Take part in a trial programme about behavioural and emotional difficultiesRead More
Introducing our Newest Trustee: Emma Simmonds
Introducing Our Newest Trustee: Emma Simmonds “On behalf of the Board of Trustees at Action Duchenne, I extend a warm welcometo Emma as our newest …
Join our NEW Online Support Groups
We decided our regular online support groups needed a bit of a re-think. To make sure we’ve got everyone covered, there are now going to be 4 …
NICE Publishes Draft Guidance on Vamorolone for the Treatment of Duchenne Muscular Dystrophy
We stand with the Duchenne community in expressing our disappointment regarding the National Institute for Health and Care Excellence's (NICE) recent …
FDA Approves Givinostat for the Treatment of Duchenne Muscular Dystrophy in the USA
Further to our previous article on the publication of the phase 3 EPIDYS trial positive results for Givinostat, a histone deacetylase (HDAC) inhibitor …
FDA Approves Givinostat for the Treatment of Duchenne Muscular Dystrophy in the USARead More
Results from Italfarmaco Pivotal Phase 3 EPIDYS Study of Givinostat in Duchenne Muscular Dystrophy (DMD) Published in The Lancet Neurology
We are thrilled to announce exciting new developments regarding the investigational drug Givinostat, after Italfarmaco S.p.A. announced today that the …
PepGen Receives UK CTA Clearance from MHRA to Begin CONNECT2-EDO51 Phase 2 Clinical Trial
PepGen, a biotech company developing innovative oligonucleotide therapies for severe neuromuscular and neurological diseases, has received approval …
PepGen Receives UK CTA Clearance from MHRA to Begin CONNECT2-EDO51 Phase 2 Clinical TrialRead More
PTC Community Statement on Translarna (Ataluren) Market Authorisation in the European Union and Great Britain
PTC has provided a community statement regarding the recently decision by the CHMP/EMA to not renew the marketing authorisation for Translarna …
