Only 37% of orphan medicines are reimbursed in Ireland. Rare Diseases Ireland calling for faster access.
Rare Diseases Ireland has highlighted a critical issue: 163 orphan medicines now have EU approval, but people living with rare diseases in Ireland face long delays getting access. They're urging the Minister for Health to establish an Early Access Scheme so patients can get life-changing treatments within one year of approval, not years down the line.
Calling All SMA Researchers: International researchers are invited to explore two exciting funding opportunities.
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.
Have you lived with a neuromuscular condition and accessed healthcare services in Ireland?
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.
The European Commission has granted marketing authorisation for SMA treatment Itvisma
The European Commission has granted marketing authorisation for Itvisma (intrathecal formulation of onasemnogene abeparvovec, developed by Novartis) for the treatment of adults and children aged 2 years and older living with 5q spinal muscular atrophy (SMA). The European Commission’s decision follows the positive opinion adopted by the Committee for Medicinal Products for Human Use (CHMP) in April 2026.
Hope for families as HSE moves to approve Duchenne drug givinostat: Prime Time speaks to campaigners and Minister for Health
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.
![[RTÉ News] 'Time is muscle' - decision urged on rare disease medicine](https://images.squarespace-cdn.com/content/v1/63c138561d3afd3c27c17d5d/1780591796791-P9MCQ6K5TEIAM4OA44CR/00246a30-800.webp)
[RTÉ News] 'Time is muscle' - decision urged on rare disease medicine
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.
![[RTÉ News] Father of rare disease patient fears losing second son](https://images.squarespace-cdn.com/content/v1/63c138561d3afd3c27c17d5d/51c88199-b155-4ab1-ad5e-dec7d8adbec8/00246f91-800.webp)
[RTÉ News] Father of rare disease patient fears losing second son
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.
Your Rare Disease Journey Matters — Share It in 30 Minutes with Rare Diseases Ireland
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.
EMA Committee Backs Novartis Gene Therapy Itvisma for SMA Patients Aged Two and Over
On 23 April 2026, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for Itvisma, a gene therapy for people aged 2 years and older living with spinal muscular atrophy (SMA).
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.