MDI have been invited to prepare a patient submission as part of the drug reimbursement assessment process for two treatments for generalised myasthenia gravis (gMG); zilucoplan (Zilbrysq) and rozanolixizumab (Rystiggo), both manufactured by UCB Pharma.  This submission is an important opportunity for people living with MG, as well as their families and carers, to share their experiences and help decision makers better understand the day-to-day realities of living with the condition. 

MDI was delighted to attend the Rare Disease Research Conference 2026 at University College Dublin, hosted by the Rare Disease Clinical Trial Network.

Children’s Health Ireland (CHI) has been recognised by Italfarmaco, the manufacturer of Givinostat, for achieving the fastest turnaround time in Europe between the treatment becoming available in Ireland and its first prescription This is a significant achievement, made possible through the determination of families who campaigned for access to the treatment, the commitment of the CHI multidisciplinary team, and collaboration with Muscular Dystrophy Ireland (MDI).

Muscular Dystrophy Ireland (MDI) is pleased to share our new research landscape review, Neuromuscular Research in Ireland: Trends, Gaps & Opportunities (1990–2025), together with an Executive Summary of its key findings.

I was delighted to receive an invite from the Minister of Health Jennifer Carol MacNeil to attend the Irish Presidency Conference on Clinical Trials and Medicine Access today. The conference is essentially about how Europe can make the journey from medical research to patient access to medicines faster, more effective and more sustainable. Looking at why there can be a significant gaps between a medicine being developed/approved and patients actually getting access to it across Europe!!

As part of Muscular Dystrophy Awareness Month, we’re delighted to highlight MDI’s successful partnership through Research Ireland’s Enterprise Partnership Postgraduate Scheme, mentoring an early-career researcher at Royal College of Surgeons Ireland (RCSI) University. This exciting partnership is helping to advance research into Duchenne muscular dystrophy (DMD) while supporting the next generation of researchers in the field. 

The National Rare Disease Office (NRDO) is pleased to launch an Expression of Interest (EOI) process seeking Patient Partners to participate in implementation of the National Rare Disease Strategy 2025-2030. Patient Partners will contribute to National Rare Disease Strategy Workstream Groups and the Patient Partnership Forum, working alongside patient advocacy organisations, clinicians, researchers, healthcare professionals, policymakers, and other stakeholders to help shape and inform implementation of the Strategy.

MDI welcomes the HSE’s decision to approve reimbursement of Skyclarys (omaveloxolone) for eligible adults living with Friedreich ataxia in Ireland. This decision comes after months of campaigning which saw the whole community come together, with people across Ireland getting behind the campaign and supporting people living with Friedreich ataxia and their families.

MDI is delighted to share an important update in our campaign for equal access to treatments for adults living with spinal muscular atrophy (SMA). 

International researchers are invited to explore two exciting funding opportunities currently available from SMA Europe and Cure SMA. Both organisations have active grant calls supporting research that aims to advance our understanding of spinal muscular atrophy (SMA) and improve outcomes for people living with the condition.

A Master's student from the School of Psychology at the University of Galway is conducting a study exploring experiences of sexual healthcare among people living with neuromuscular conditions in Ireland. 

Families affected by Duchenne muscular dystrophy received welcome news this week, as the HSE drugs group recommended approval of givinostat, a treatment that can help slow the progression of the condition.

The parents of a child with a rare muscular disease are urging that the State does "not delay any longer" in making decisions on access to a drug that has been approved for use in Europe but has not been approved for reimbursement in Ireland.

Patients with rare diseases and their families travelled to Leinster House to call for action on access to new medicines.The group is calling on the Government to introduce a dedicated new line item in the Budget for new drugs, a review of the reimbursement scheme and an expansion of the Early Access Programme.

Rare Diseases Ireland is testing a new questionnaire designed specifically to capture the healthcare experiences of people living with rare diseases and their caregivers. The questionnaire was built specifically for rare diseases - covering the things standard questionnaires always miss, like how long it took to get a diagnosis, or what it's like coordinating care across multiple specialists.