Family Science Education Day and Charity Dinner Dance Join us for a Family Day at Birmingham's Think Tank Science Museum followed by a Charity …
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Action Duchenne launches Webinar Series 2025
Action Duchenne launches Webinar Series 2025 Action Duchenne brings a great initiative to further support the Duchenne community through their …
Action Duchenne Expands All-Through Support into Scotland
Action Duchenne Expands All-Through Support into Scotland PRESS RELEASE FOR IMMEDIATE RELEASE: Action Duchenne to deliver vital support to …
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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is excited to share the news that the National Institute for Health and Care Excellence (NICE) has issued final draft guidance …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
Empowering lives on International Day of Persons with Disabilities
As we mark International Days of Persons with Disabilities today, 3rd December, we are delighted to share significant milestones in our ‘All-through …
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Givinostat Early Access Programme Launches for Duchenne Muscular Dystrophy Patients
Summary We are excited to announce that Givinostat, a new HDAC (histone deacetylase) inhibitor treatment currently under regulatory review, is now …
Givinostat Early Access Programme Launches for Duchenne Muscular Dystrophy PatientsRead More
Winter Webinar – NICE & Drug Approval
To kick start out Winter Webinars we will be joined by the National Institute for Health and Care Excellence (NICE) who will explain their role in the …
