Roche and Sarepta Update on ELEVIDYS Study This week Roche and Sarepta Therapeutics have shared updates on the ongoing development programme …
Sevasemten: Servier shares Clinical Trial Results in Becker Muscular Dystrophy
On 3rd November, Servier announced the Phase 3 GRAND CANYON clinical trial of sevasemten in people living with Becker muscular dystrophy (BMD) did not …
Sevasemten: Servier shares Clinical Trial Results in Becker Muscular DystrophyRead More
Givinostat (Duvyzat®): MHRA Shares Interim Monitoring Advice
On 5 November 2026, the Medicines and Healthcare products Regulatory Agency (MHRA) published interim advice for healthcare professionals and families …
Givinostat (Duvyzat®): MHRA Shares Interim Monitoring Advice Read More
Entrada Provides Update on Clinical Trial Progress
Entrada Therapeutics has released its latest quarterly update on its Duchenne clinical trial programmes. Below, we've summarised what's new for each …
Science Education Workshops: Decoding Duchenne Together
Science Education Workshops: Decoding Duchenne Together This Summer our fun filled family days will also offer you the opportunity to …
Science Education Workshops: Decoding Duchenne TogetherRead More
Halfway Through Action Duchenne’s Webinar Series 2026: Learning, Connecting and Growing Together
Halfway Through Action Duchenne’s Webinar Series 2026: Learning, Connecting and Growing Together "The support our family has received from …
Solid Biosciences Shares Update on SGT-003 Duchenne Gene Therapy Programme
Solid Biosciences has shared an update on SGT-003, its investigational gene therapy for Duchenne muscular dystrophy (DMD), coinciding with …
Solid Biosciences Shares Update on SGT-003 Duchenne Gene Therapy ProgrammeRead More
We’re Recruiting: Director of Services and Safeguarding
We're Recruiting: Director of Services and Safeguarding Welcome to Action Duchenne “Your child has Duchenne Muscular …
We’re Recruiting: Director of Services and SafeguardingRead More
Translarna (Ataluren) Not Accepted for Continued Use Under NHSScotland
The Scottish Medicines Consortium (SMC) has completed a reassessment of Translarna (ataluren) and has decided not to recommend it for routine use …
Translarna (Ataluren) Not Accepted for Continued Use Under NHSScotlandRead More
Make a real difference with our amazing events and challenges
“I just want to say that Victoria and the rest of the Action Duchenne family are incredible human beings. The support they give is amazing. When you …
Make a real difference with our amazing events and challengesRead More
Entrada Therapeutics Provides Quarterly Update on ELEVATE Studies.
Entrada Therapeutics has shared its latest quarterly update across all four of its active Duchenne programmes. For families in the UK, there are …
Entrada Therapeutics Provides Quarterly Update on ELEVATE Studies. Read More
NICE Approves Givinostat for NHS Use in England
We are delighted to share the news that the National Institute for Health and Care Excellence (NICE) has approved Givinostat (Duvyzat) for use within …
