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
Edgewise Therapeutics Announces Positive Results from the CANYON Phase 2 Trial of Sevasemten in Individuals with Becker Muscular Dystrophy
Edgewise Therapeutics, announced positive topline results from the Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy. …
NICE Recommends Vamorolone for Treating DMD in England
Action Duchenne is excited to share the news that the National Institute for Health and Care Excellence (NICE) has issued final draft guidance …
NICE Recommends Vamorolone for Treating DMD in EnglandRead More
REGENXBIO Announces Pivotal Phase of AFFINITY DUCHENNE Clinical Trial for RGX-202
REGENXBIO, a leading clinical-stage biotechnology company focused on advancing transformative gene therapies, announced in late November that the …
REGENXBIO Announces Pivotal Phase of AFFINITY DUCHENNE Clinical Trial for RGX-202 Read More
