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
Entrada Therapeutics: 2026 Clinical Trial Plan & Progress
Entrada Therapeutics has shared several key milestones regarding their ELEVATE clinical trial programs. These studies are designed to evaluate the …
Entrada Therapeutics: 2026 Clinical Trial Plan & ProgressRead More
Sarepta Announces Topline Three-Year EMBARK Results: Long-Term Performance of ELEVIDYS Gene Therapy
Sarepta Therapeutics has released updated findings from its Phase 3 EMBARK study, providing a three-year assessment of the gene therapy ELEVIDYS …
Training Plans and Injury Prevention
If you have signed up for an event and registered on the official race console, you will receive email communications from the event. This will …
