Clinical Trial Update: Early Results Published for DM1 Investigational Therapy
Pharmaceutical company PepGen has published preliminary results from the first phase of its FREEDOM2-DM1 clinical trial, which is investigating a potential new therapy for people living with myotonic dystrophy type 1 (DM1).
Campaign for Friedreich's Ataxia Treatment Access Continues — Read the Latest
FARA Ireland's April 2026 Advocacy Bulletin details the latest steps being taken to secure reimbursement of SKYCLARYS for people living with Friedreich's Ataxia in Ireland. Download the full bulletin for an update on where things stand.
Minister for Health announces introduction of screening for Severe Combined Immunodeficiency and Spinal Muscular Atrophy
Muscular Dystrophy Ireland warmly welcomes the introduction of testing for spinal muscular atrophy (SMA), commencing today as part of the expansion of the National Newborn Bloodspot Screening Programme
Clinical Trial Updates from the USA on Becker Muscular Dystrophy and Duchenne Muscular Dystrophy
A lot has happened recently regarding clinical trials for Duchenne Muscular Dystrophy, and Becker Muscular Dystrophy (Hereafter referred to as Duchenne and Becker). All of these updates are from the American Muscular Dystrophy Association Clinical & Scientific Conference in Orlando that took place from March 8th - 11th let’s start with an update from a Becker Trial.
NCPE Completes Givinostat Review: What It Means for Duchenne Families
In November, MDI, in collaboration with Duchenne families, launched a survey to capture the lived experience of families living with Duchenne muscular dystrophy to support the patient submission for the reimbursement of givinostat. We would like to say a heartfelt thank you to everyone who took the time to participate.
Share Your Voice: Rare Disease Family Cost Diaries: Economic Impact
This project forms part of a wider research study examining the social and economic impacts of living with a rare disease on families of children with rare diseases across Ireland and Northern Ireland. The research is being conducted as part of a PhD project by researchers at University College Dublin.
Duchenne Muscular Dystrophy (DMD) Paediatric and Adult Care Pathway Published
The pathway outlines recommendations for the key specialists and services involved in DMD care, supporting coordinated and consistent care for children, young people and adults living with DMD.
Access to Orphan Medicines
MDI provide a written submission to the Joint Committee on Health on issues relating to rare diseases, including access to and reimbursement for medicines.
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.
Help Improve Cancer Care for Individuals with Physical Disabilities
Researchers at DCU are examining the supportive care needs of people with pre-existing physical disabilities who are diagnosed with cancer.
Invitation to participate in a research study on Community-Based Healthcare for Individuals with Neuro-Physical Disabilities
Have your say on what matters most when accessing and using community-based healthcare services. A PhD Researcher in Health Economics at University College Cork, is exploring how community-based healthcare services can better support people living with neuro-physical disabilities. The results will give a clearer picture of how to design community-based care that truly meets the needs of people with neuromuscular and other neuro-physical conditions. This evidence will help guide public policy, reduce inequalities, and improve social wellbeing and sustainability.
Online Information Webinar - Improving Access to Clinical Trials
As part of IPPOSI’s Patient Capacity Building Programme, IPPOSI is delighted to host an online information webinar in collaboration with ClinicalTrials.eu – a new European Union platform designed to make clinical trial information more accessible to the public.
MDI Open Call: HRCI-HRB Joint Funding Scheme 2025/2026 Expressions of Interest
MDI is pleased to invite expressions of interest for the 2025/2026 HRCI‑HRB Joint Funding Scheme.
The Psychosocial Needs of Adults Living with Muscular Dystrophy and Similar Neuromuscular Conditions in Ireland.
In 2024, Dr. Finiki Nearchou and her team at UCD, in partnership with MDI, completed a study on the psychosocial needs of adults with muscular dystrophy and similar neuromuscular conditions.
‘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.
Update on Duvyzat™ (givinostat) for Duchenne Muscular Dystrophy
Duvyzat™ receives positive recommendation from the EMA’s CHMP for conditional approval in DMD.
Click to read what this could mean for families and what happens next.
The Socio-Economic Impact of Living with a Rare Disease for Families of Children with Rare Diseases
Caring for a child with a Rare Disease comes with financial, social, and emotional challenges. Share your experiences in a 30-min survey. Your voice matters—help shape better support systems.
🧠 HSE Project: Improving Care for People with a Neurological Condition
The HSE is working on a new plan (called an integrated approach) to help improve care for people with a neurological condition. A neurological condition is when the brain, spinal cord, or nerves aren’t working properly. Some examples include Parkinson’s disease, epilepsy, head trauma, multiple sclerosis, motor neurone disease, headache (and migraine). We want to hear from people who use these services—patient and service user partners — so we can help improve care together.
Strengthening Global Ties: MDI Joins FSHD Europe, Advances Registry Work, and Supports Patient Voice in Access to Treatments
MDI has joined FSHD Europe, a key alliance supporting patients with facioscapulohumeral muscular dystrophy across the continent, aiming to improve diagnosis, care, and treatment access. We also recently took part in the TREAT-NMD Annual Curators Meeting, gaining valuable insights as we continue to develop a national neuromuscular registry for Ireland. Lastly, MDI highlights the role of patient organisations in Health Technology Assessments (HTAs) — a crucial step in securing access to new treatments. We may seek patient input for upcoming HTA submissions, so stay tuned for future opportunities to share your voice.