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 …
We’re Recruiting: Outreach Support Officer (Scotland)
Outreach Support Officer (Scotland) Role: Outreach Support Officer (Scotland)Responsible to: Director of ServicesPay Band: Band 3, point 1 (£30,799 …
We’re Recruiting: Outreach Support Officer (Scotland)Read More
We’re Recruiting: Events Officer
Events Officer Role: Events OfficerResponsible to: Director of ServicesHours: 0.6 FTE (22.5 hours per week)Pay Band: Band 3, point 1 (£30,799 …
Sarepta Provides Regulatory Update on AMONDYS 45 and VYONDYS 53
Sarepta Therapeutics has announced that, following completion of the ESSENCE confirmatory study and a period of engagement with the U.S. Food and Drug …
Sarepta Provides Regulatory Update on AMONDYS 45 and VYONDYS 53Read More
Edgewise Therapeutics Announces Long-Term Sevasemten Data in Becker Muscular Dystrophy
Edgewise Therapeutics has presented long-term data from the MESA study of sevasemten, an investigational treatment for Becker muscular dystrophy, at …
Edgewise Therapeutics Announces Long-Term Sevasemten Data in Becker Muscular DystrophyRead More
REGENXBIO Reports New Positive Interim Data from AFFINITY DUCHENNE Trial
REGENXBIO has shared encouraging new data from their ongoing Phase I/II AFFINITY DUCHENNE trial of RGX-202, an investigational gene therapy for …
REGENXBIO Reports New Positive Interim Data from AFFINITY DUCHENNE TrialRead More
Update on givinostat NHS England decision – and what it means for families
The decision on whether Duchenne muscular dystrophy treatment, givinostat, can be offered on the NHS in England for patients aged 6 years and older …
Update on givinostat NHS England decision – and what it means for familiesRead More
Independent Data Monitoring Committee Recommends Increased Dose in ELEVATE-44-201 Study
Entrada Therapeutics has shared encouraging news from its ELEVATE-44-201 clinical study. An independent Data Monitoring Committee (DMC) has completed …
Independent Data Monitoring Committee Recommends Increased Dose in ELEVATE-44-201 StudyRead More
Solid Biosciences Provide SGT-003 Gene Therapy Trial Updates
In its January 2026 report, Solid Biosciences shared an update on SGT-003, our investigational next-generation gene therapy for Duchenne muscular …
Solid Biosciences Provide SGT-003 Gene Therapy Trial UpdatesRead More
