Roche have provided an update on the latest top-line results from the second year of their EMBARK Phase 3 clinical trial (NCT05096221) investigating …
Roche Shares Top-line Results from Year Two of the EMBARK Phase 3 Clinical Trial.Read More
Calling all Siblings and Caregivers of Individuals with Duchenne
Help researchers and mental health professionals at the University of Newcastle better understand how Duchenne muscular dystrophy (DMD) impacts …
Calling all Siblings and Caregivers of Individuals with DuchenneRead More
Vamorolone for Duchenne Muscular Dystrophy: A Summary of Evidence So Far
DMD Care UK will host a webinar to summarise the evidence for vamorolone as a treatment for Duchenne muscular dystrophy (DMD), and provide an …
Vamorolone for Duchenne Muscular Dystrophy: A Summary of Evidence So FarRead More
Open Letter from the Chair and CEO of Action Duchenne for Rare Disease Day 2025
Dear Friends, Supporters, and Advocates,On Rare Disease Day 2025, we stand together to raise awareness, celebrate our community, and drive meaningful …
Open Letter from the Chair and CEO of Action Duchenne for Rare Disease Day 2025Read More
Solid Biosciences Shares 90 Day Update From SGT-003 Gene Therapy Trial
We're excited to share news that Solid Biosciences has released initial data from its Phase 1/2 INSPIRE DUCHENNE clinical trial, assessing the safety …
Solid Biosciences Shares 90 Day Update From SGT-003 Gene Therapy TrialRead More
Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44
Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44Entrada Therapeutics, today announced that the United States Food and Drug …
Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44Read More
Family Science Education Day and Charity Dinner Dance
Family Science Education Day and Charity Dinner DanceJoin us for a Family Day at Birmingham's Think Tank Science Museum followed by a Charity Dinner …
Family Science Education Day and Charity Dinner DanceRead More
Action Duchenne launches Webinar Series 2025
Action Duchenne launches Webinar Series 2025Action Duchenne brings a great initiative to further support the Duchenne community through their Webinar …
Action Duchenne Expands All-Through Support into Scotland
Action Duchenne Expands All-Through Support into ScotlandPRESS RELEASEFOR IMMEDIATE RELEASE:Action Duchenne to deliver vital support to Scottish …
Action Duchenne Expands All-Through Support into ScotlandRead More
SMC Recommends Vamorolone for Treating DMD in Scotland
Action Duchenne welcomes the exciting news from the Scottish Medicines Consortium’s (SMC) positive recommendation for vamorolone for the treatment of …
SMC Recommends Vamorolone for Treating DMD in ScotlandRead More
Merry Christmas and Happy New Year from Action Duchenne
MHRA approves Givinostat as a treatment for Duchenne muscular dystrophy
Action Duchenne is pleased to share great news with the Duchenne community. Givinostat has been approved by the Medicines and Healthcare products …
MHRA approves Givinostat as a treatment for Duchenne muscular dystrophyRead More
