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is excited to share the news that the National Institute for Health and Care Excellence (NICE) has issued final draft guidance …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 …
Action Duchenne Annual International Conference 2024: Welcome Remarks
It is so lovely to see so many familiar faces here, to meet new ones as well. My name is Florence Boulton. I’m the CEO of Action Duchenne. I joined …
Action Duchenne Annual International Conference 2024: Welcome RemarksRead More
Sarepta Announces the Discontinuation of MOMENTUM Study and Development of SRP-5051
We are disappointed to learn of the news regarding Sarepta’s SRP-5051 MOMENTUM study. Sarepta has decided to stop developing SRP-5051, a potential …
Sarepta Announces the Discontinuation of MOMENTUM Study and Development of SRP-5051Read More
Charity Dinner Dance
Charity Dinner Dance In March 2023, Ruth Taylor and her amazing committee members held an incredible Charity Dinner Dance to fundraise for Action …
Annual General Meeting: Friday 8th November 2024
Annual General Meeting 2024 The Trustees of Action Duchenne would like to express their gratitude for the continued support from the Duchenne …
Help Develop Nutrition Information Resources for Duchenne Muscular Dystrophy
If you are a caregiver of/or a young person aged 7-25 with Duchenne, researchers from the University of Glasgow, and Edge Hill University, need your …
Help Develop Nutrition Information Resources for Duchenne Muscular DystrophyRead More
Wave Life Sciences Announces Positive Interim Data from FORWARD-53 Clinical Trial Evaluating WVE-N531 in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
Wave Life Sciences has shared their promising new interim results for the ongoing FORWARD-53 trial. The ongoing open-label trial, involves eleven boys …
