As we mark International Days of Persons with Disabilities today, 3rd December, we are delighted to share significant milestones in our ‘All-through …
Empowering lives on International Day of Persons with DisabilitiesRead More
Givinostat Early Access Programme Launches for Duchenne Muscular Dystrophy Patients
SummaryWe 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 DanceIn 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 2024The Trustees of Action Duchenne would like to express their gratitude for the continued support from the Duchenne community …
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 …
Swissmedic Announces Acceptance of Santhera’s Market Authorisation Application for AGAMREE (Vamorolone) in Duchenne Muscular Dystrophy
Santhera Pharmaceuticals (SIX: SANN) has announced that Swissmedic, the Swiss Agency for Therapeutic Products, has begun reviewing the marketing …
Action Duchenne: Delivering Hope & Change in 2024
We are pleased to share our Impact 2024 report, which highlights our progress and achievements over the past year. It showcase the hard work and …
Together for World Duchenne Awareness Day: Celebrating Strength and Progress
"On World Duchenne Awareness Day 2024, we come together as a community—families, friends, carers, and supporters—alongside everyone living with …
Together for World Duchenne Awareness Day: Celebrating Strength and ProgressRead More
