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.
There is still time to Move for MDI this month! 🚴♀️
Find out why Cormac is moving for MDI and join him in whatever way works for you! Sign up on our campaign page.
Rathfeigh, Skryne & Tara community raise €1,113 for MDI
The Rathfeigh, Skryne & Tara community chose MDI as their charity of choice for their recent Family Field Day. The event was a huge success with a barbeque and fun family activities and raised an incredible €1,113.04 which was received with thanks by MDI.
Inclusion of children and young people in clinical trials across Europe
The aim of this group is to facilitate the inclusion of children and young people in clinical trials across Europe avoiding any language barriers. The group recently identified that some patients could not take part in a clinical trial because they do not speak the official language of the country where the trial is performed.
Spotlight on members of our community living with neuromuscular conditions: Oisín Quinn
As part of our members’ stories series, 13-year-old Oisín Quinn is delighted to share his story about his interest in music. Oisín lives with the neuromuscular condition, Charcot Marie Tooth type 1X (CMT1X).
Advocating for people living with neuromuscular conditions and their families:MDI and our umbrella organisations – Budget 2025 asks and general election manifestos.
We provide an overview of key issues identified by MDI as important for our community and a flavour of how some of these umbrella organisations are championing these causes in their pre-budget 2025 submissions and election manifestos. We are members of Care Alliance Ireland, Disability Federation of Ireland, Health Research Charities Ireland (HRCI),The Irish Platform for Patient Organisations, Science & Industry (IPPOSI), Neurological Alliance of Ireland (NAI), Rare Diseases Ireland (RDI) and The Wheel. You can read their specific submissions/manifestos by clicking on each of the organisations’ names above.
Join Paracyclist Cormac O'Callaghan and Move for MDI
Whether you walk, roll, play boccia, cycle - we want you to move in any way that feels good to you, your participation will help support those living with muscular dystrophy across Ireland. Every movement makes a difference!
Breaking Down Barriers: University of Galway and Chronic Pain Ireland Event on Sex and Gender in Chronic Pain
This hybrid event will explore the critical issue of sex and gender in chronic pain, inviting all genders to join the conversation. While chronic pain may affect individuals differently, this event will outline what we now know about sex and gender as variables in chronic pain, what impact the outcomes of the PAINDIFF network will have on the study of sex and gender in pain and how this may inform on diagnosis and treatment outcomes for everyone.
MDI is a proud member of Health Research Charities Ireland and supports the HRCI manifesto
HRCI members demand a strong, forward-thinking Programme for Government which includes a genuine commitment to improving health outcomes and reducing the burden of illness. Investing in health research (including life sciences, clinical research and all forms of R&D in health) is a commitment to the well-being of the population, the advancement of medicine and care, and the overall prosperity of our society.
September 30th is Limb Girdle Muscular Dystrophy Awareness Day!
Limb Girdle Muscular Dystrophy Awareness Day is an annual collaborative effort to globally raise awareness of individuals living with limb girdle muscular dystrophy (LGMD).
Neurological Alliance of Ireland Pre Budget Submission
As a member organisation of the Neurological Alliance of Ireland, MDI supports its pre-budget submission which calls for funding to begin to tackle the regional inequity in access to neurology and neurorehabilitation services. Find out more here. We also support the NAI’s election manifesto.
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.
September 25th is World Ataxia Awareness Day
International Ataxia Awareness Day (IAAD) is on September 25th of each year. It is a coordinated effort from individuals and Ataxia organizations around the world to help shed light on this rare disease. NAF is committed to leading the way on this outreach effort – but we need your help! We encourage individuals and disease organizations to join us by planning events and/or awareness campaigns for IAAD.
Today is World Myositis Day!
Myositis is an inflammatory autoimmune condition that can cause muscles to become weak and painful. Idiopathic inflammatory myopathies are an umbrella term for the different types of myositis. These autoimmune conditions mean the body attacks its own muscles, connective tissue, blood vessels, skin, lungs, and other organs. This often occurs together with other symptoms, such as muscle swelling, pain, fatigue, difficulty moving limbs or lifting arms, increased falls, trouble swallowing, and others.
Darts Fundraiser and Half Marathon Raise Over €4,900 for MDI with World-Class Darts Stars
Stepanie & John O’Hara go all out every year to bring world-class players from the darts world to Ireland and this year was the turn of Simon Whitlock, who gave his jersey to the lovely Shannon at the end of the night! This year’s events raised €1,884.90 to support our work.
This Week is World Mitochondrial Awareness Week!
World Mitochondrial Disease Week raises awareness about mitochondrial diseases (mito). It aims to improve the lives of people affected by mito and to increase awareness among doctors and the general public.
![[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!
Be one of the first 10 households to book your spot at MDI’s Conference and receive an Amazon Alexa for attending!
Join us for Muscular Dystrophy Ireland's National Conference, Living life to the fullest, on 10 November 2024 at Croke Park, Dublin! This day-long event offers a supportive space for people with neuromuscular conditions (NMCs) and their families to connect, share and learn.
Spotlight on members of our community living with neuromuscular conditions: Rose Maloney Quinn
As part of our members’ stories series, Rose Maloney Quinn is delighted to share her story about her career. Rose lives with the neuromuscular condition, Charcot Marie Tooth type 1X (CMT1X).
Today Is International Myotonic Dystrophy Awareness Day!
The colour green has been chosen to represent International Myotonic Dystrophy Awareness Day which takes place on 15 September and many buildings throughout the world will be lit up green on the day! As MDI’s News Update focused on Myotonic dystrophy last January, we are happy to provide an update on research developments since then.