Rare disease is an issue that affects thousands of families across Ireland, yet too often it receives far too little attention in this House. In particular, many patients face struggles gaining access to the medicines and treatments that could transform or even save their lives. The word "rare" can be misleading. When people hear the term “rare disease”, they imagine something that affects only a handful of people, but when we put all of these conditions together, the reality is very different. Today, around one in every 17 people in Ireland is living with a rare disease. That means hundreds of thousands of people across the country are affected in some way. Behind every statistic is a family. There is a child waiting for a diagnosis. There is a parent fighting to access treatment. There is a young adult wondering whether their condition will deteriorate before a medicine becomes available, and there are families carrying enormous emotional, physical and financial burdens while having to navigate a healthcare system that is often difficult to access and even harder to understand. A treatment may exist. A medicine may already be approved elsewhere in Europe. Patients may know that the drug could help them, yet they can still face lengthy delays before that medicine becomes available in Ireland. I particularly want to acknowledge the families who have spent months and years fighting for access to givinostat for children living with Duchenne muscular dystrophy. The recent recommendation from the HSE drugs group to approve this treatment is very welcome news and a testament to the determination of parents who simply refused to accept that their children should be left waiting while the clock ticked on. These families have shown enormous courage and persistence. They have attended meetings, spoken to public representatives, told their stories in the media and campaigned relentlessly because they understood one simple truth — time is muscle. Every day matters when a child is living with Duchenne. I welcome the decision by the senior HSE leadership team to approve this treatment following intensive lobbying by Muscular Dystrophy Ireland, affected families and patient advocates. It shows what can be achieved when people come together and refuse to give up. We must now ensure that eligible children can access this medicine without delay and that the lessons learned from this campaign are applied to other rare disease treatments in the future. I am calling on the Minister to ensure that the managed access protocol is expedited for these families. Independent Ireland believes that every citizen should have access to healthcare based on need, not on where they live, who they know or how effectively they can campaign for attention. We believe that patients suffering from rare diseases deserve the same urgency and commitment from the State as patients with more common conditions. The publication of Ireland's national rare disease strategy was an important and welcome step. It recognises the need for earlier diagnosis, better care pathways, improved research, stronger patient involvement and improved access to orphan medicines, but strategies alone do not change lives. Implementation changes lives. Families do not need another glossy document sitting on a shelf. They need action, appointments, diagnostics and, where appropriate, they need timely access to treatment. The current system often evaluates these medicines using frameworks designed for large population treatments. As a result, patients can experience lengthy delays before decisions are made. Independent Ireland believes there is room for a more flexible and patient-centred approach. Our party believes that where a medicine has demonstrated clear clinical benefit and has received European regulatory approval, every effort should be made to ensure that Irish patients are not left waiting unnecessarily while administrative processes continue. Independent Ireland has consistently argued that public services should be built around the needs of citizens rather than bureaucracy. The healthcare system is no different. The needs of patients and families must come first. Recent agreements aimed at accelerating access to new medicines are welcome. However, the true test will not be what is written in an agreement; but whether patients and families notice a difference in their everyday lives. Can a child receive treatment sooner? Can a family avoid travelling abroad? Can a patient access a medicine before irreversible damage occurs? Those are the questions that matter. Independent Ireland has consistently highlighted the challenges faced by families living outside major urban centres. Many rare disease patients must travel repeatedly to Dublin and to specialist centres for consultations, diagnostics and treatment. The financial burden, the time away from work and school and the emotional strain this places on families can be enormous. We need faster diagnostic pathways. We need improved support services for families. We need better regional access to specialist care wherever possible. We need a medicines approval system that recognises the particular challenges associated with rare diseases. More importantly, we need to listen to patients. Nobody understands the shortcomings of the system better than those who live with these conditions every day. Their voices must be at the centre of decision-making. A society is often judged by how it treats those who are most vulnerable. Families affected by rare diseases have shown extraordinary resilience, courage and determination. They should not have to spend years fighting for recognition, they should not have to spend years fighting for treatment and they certainly should not have to spend years fighting bureaucracy while their conditions deteriorate. Our job in this House is to ensure that hope is matched by action. That is what fairness demands. That is what compassion requires. That is what Independent Ireland believes should be delivered. I wish to ask the Minister of State about Friedreich's ataxia and the medication Skyclarys. Where are we in relation to getting patients access to Skyclarys? The brother of Emma O'Shea in west Cork died from this disease. I met a group of young people who were here in Leinster House a few weeks ago. One was a young lady who was devastated by what the disease was doing to her. Every second without Skyclarys medication is a death sentence. I ask the Minister of State, how can we in opposition help the Government? We are not here to blame the Government, but how can we help? Surely if one of our children needed Skyclarys, we would find a way. I ask the Minister of State to please tell me how we can help. In his summing up, he might let me know where developments with regard to Skyclarys are. I have been in contact personally with Emma O'Shea continuously, even just up to a few minutes ago. She is struggling greatly and wants to know when there will be an announcement. I am not here to point fingers; I am just here to plead on behalf of that lady and other people who need Skyclarys. I would really appreciate when the Minister of State is summing up if he could give us some guideline. There was talk it might be announced last week - that did not happen - and that it could be announced next month. If it is, we would be absolutely overjoyed. This is a life-and-death issue. If Emma lost her brother, it is quite possible she could lose her own life. She is determined to fight on. There was a very strong piece in the Southern Star today about Emma and what she was suffering and going through. I would really appreciate if the Minister of State would give us some idea as to where the process is and whether there is a possibility in that regard. It might be clearer next month. If it is, it will certainly lead to saving Emma's life and the lives of many more out there.

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