Share Your Experience: Help Inform Access to Givinostat for Duchenne Muscular Dystrophy in Ireland
A review is under way to decide if Givinostat for Duchenne muscular dystrophy will be reimbursed in Ireland. Your experience of living with Duchenne can help inform this decision - share your story in the survey link.
The World Duchenne Awareness Day 2025 theme is “Family: the heart of care”
World Duchenne Awareness Day (WDAD) is 7 September, which highlights the role of family members for people living with Duchenne and Becker muscular dystrophy. Living with Duchenne muscular dystrophy (DMD) is a journey marked by both physical challenges and emotional resilience. At the centre of this journey is family.
‘Time Is Muscle’: MDI Supports Families in Campaign for Rapid Review of Duchenne Treatment Givinostat
Please join us in asking your local TDs and senators to keep this matter on the political agenda by attending a briefing on the campaign at the Audio-Visual Room, Leinster House at 2.30pm on Wednesday 11 June. and leveraging your role as you elected representative to highlight the importance of timely and planned action. Time is muscle and delays in reimbursement risk excluding eligible children simply because the disease progresses while they wait.
EMA Recommend Against Renewing Translarna for DMD
The Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency, has again advised against renewing approval for Translarna (ataluren), a drug used to treat Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.
Brain Involvement in Dystrophinopathies (BIND) Project
The BIND project is the first large-scale effort to better understand brain involvement in Duchenne and Becker Muscular Dystrophy (DMD and BMD). Funded by the EU, it brings together 19 partners from Europe and Japan. While muscle weakness has been the primary focus in recent decades, brain involvement has received less attention.
![[Watch] Raise your voice for Duchenne: the documentary for World Duchenne Awareness Day 2024](https://images.squarespace-cdn.com/content/v1/63c138561d3afd3c27c17d5d/1726589633737-UYGRVKF0P4KF83C4JUKF/GSRvM8vXYAAdLOH.jpg)
[Watch] Raise your voice for Duchenne: the documentary for World Duchenne Awareness Day 2024
September 7 is World Duchenne Awareness Day. On this day we raise awareness for Duchenne and Becker muscular dystrophy around the globe. As every year, World Duchenne Awareness Day have a special theme that deserves more attention. This year saw the official launch of the documentary Raise Your Voice for Duchenne, directed by Nicoletta Madia and produced by Arim Communication!
7 September is World Duchenne Awareness Day
On 7 September the World Duchenne Organisation will launch a documentary that portrays the lives of people living with Duchenne muscular dystrophy across the globe, it will share their journeys, challenges and successes, and captures the resilience and determination of the Duchenne community. People are encouraged to share it with their wider communities.
Spotlight on members of our community living with neuromuscular conditions: David Kennedy
As part of our members’ stories series, we are delighted to share David Kennedy story about the benefits he gains from keeping active through swimming.
CHMP Issues Negative Opinion on Translarna™ Following European Commission Request for Review
EMA’s human medicines committee (CHMP) has recommended non-renewal of the conditional marketing authorisation of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy in Europe. This opinion follows the return of the previously issued negative opinion by the European Commission for re-review. Click here to read PTC Therapetics press release.
Spotlight on members of our community living with neuromuscular conditions: Alex James Kennedy
As part of our members’ stories series, we are delighted to share Alex James Kennedy’s story about his music career.
Watch: MDI Duchenne Muscular Dystrophy Community Gathering (Dublin, 18.11.2023)
We are happy to share our recording of our Duchenne muscular dystrophy community gathering which took place online and at MDI in Dublin on 18 November 2023. This is part of our series of condition specific community gatherings that aims to facilitate people living with specific conditions and their families to get together as a community and learn together.
![[Watch]: The tech helping teen with Duchenne muscular dystrophy live his musical dream | BBC News](https://images.squarespace-cdn.com/content/v1/63c138561d3afd3c27c17d5d/1712761630243-244S5THUMRQ8OL9UCWTV/Screenshot+2024-04-10+160654.png)
[Watch]: The tech helping teen with Duchenne muscular dystrophy live his musical dream | BBC News
An assistive technology has now given the 16-year-old the potential to keep playing music longer. And he has been given the chance to perform at America’s music, tech and film festival.
Action Duchenne: Winter Webinar – James Poysky
As part of Action Duchenne’s: Science Education programme, they will be running a series of Winter Webinars with some of your favourite Duchenne experts! Action Duchenne are delighted to announce that they will be welcoming renowned psychologist and Duchenne expert Dr James Poysky to theiir webinar series.
Round Up On Research Developments By Condition
Neuromuscular conditions can be difficult to diagnose and manage, so research is vital for families and for quality of life. In this issue, we include a round-up of some research developments that have taken place in recent months