I acknowledge that the Minister is here in person. It does not always happen for Topical Issues but I am grateful for the fact that she is here. It is probably a reflection of the fact that she is getting the same emails I am getting from people in our constituency who are affected by Duchenne muscular dystrophy. I am sure the Minister already knows about it but Duchenne muscular dystrophy is a condition that affects muscular restoration and muscular development, mostly in boys although not exclusively. It occurs from about the age of four. It is a degenerative condition. Over time, it restricts the ability of those boys to do things that all our children take for granted, simple things, whether that is getting up after a fall, getting up out of a chair, getting in and out of a car or climbing stairs. Over time, that gets worse and worse until eventually they cannot stand independently or cannot stand up at all. Unfortunately, the nature of this condition is that as it gets worse and worse, boys who suffer from it will often not be able to walk by the age of 12. This is, therefore, a heartbreaking condition for parents as they watch their young children unable to do the things their peers are able to do. That degenerative nature of the disease must be extraordinarily difficult for them. It is a rare disease. I know there are different assessments as to how common it is. Ultimately, it does not affect a huge number of people but those people whom it does affect are deeply affected by it, not only the families and the extended families but also, obviously, the people who suffer from it themselves. The Minister is aware that families were in here last week, a year after they first brought this to our attention, to have a meeting to inform many of the Members of this House about the situation in relation to Duchenne muscular dystrophy and the treatments that are available. Many Members will have attended that briefing in the audiovisual room. The families referred specifically to givinostat, a drug that is available for the treatment of the condition. Givinostat is really important because it is a turning point in the treatment of Duchenne muscular dystrophy. It cancels out some of the enzymes that prevent muscular development and muscular restoration. The muscles of young boys who suffer from Duchenne's are generally wasting away; they cannot restore themselves. Now there is a drug that is available and has been approved, for example, in the United States and provisionally in the European Union and that can treat that specific cause and deal with the enzyme that prevents the muscles from regenerating and being restored. It acts as a pause or a stop on the progress of the disease. There are a couple of difficulties with it, however. The first is that it is not something that can be taken at any stage in a sufferer with Duchenne's. There are boys who are no longer eligible to take givinostat because of the delays in getting the drug approved in this jurisdiction. There are other boys who are approaching the point at which they will not be able to take it. Time is therefore of the essence in this matter. As one parent who wrote to me said, time is muscle when it comes to Duchenne's. Again, I am sure the Minister is familiar with these issues. Those parents who have children who suffer from Duchenne's are therefore incredibly anxious that this drug will progress. I know that was a meeting - last week, I think - of the senior management team or that this is progressing through the Minister's Department. What I would really like her to tell us, though, is the progress in relation to givinostat; whether the families can expect that this drug will be available not only to them but on the same terms that it is available in other parts of the island - for example, in Northern Ireland; and if she can tell parents, conscious that time is muscle and conscious that there are kids who need this drug urgently, if this will progress in such a way that it will be available to them in the short term, or when it will be available to them. This heartbreaking disease can be stopped in its tracks for some patients, and we should be doing everything we can to ensure that happens.

Sentiment score: -0.07

I thank the Minister. I join with her in thanking the HSE for the work that has been done and also acknowledge her commitment to this. Anybody who has dealt with families who are suffering with this disease understands both the heartbreaking nature of it and the urgency involved. Listening to what the Minister has said in relation to the progress and the managed access protocol, do we have a timeline for when it will be available? I accept entirely what the Minister has said about the clinical criteria for the application of the drug. That is entirely appropriate and, unfortunately, there will be boys, because of the delay in the application outside our control, who may not be eligible to receive the drug now. For those who are still eligible and those who can still stand up independently or who can stand up, can we give them a timeline for when they might be assessed for that and the conditions under which it will be made available to them? I am conscious of the fact that muscle is time and the fact that this drug is the very best way available to sufferers of Duchenne muscular dystrophy to stop the progress of this disease and to stop the degeneration of their muscles, which is irreversible. For the families the Minister has been dealing with and I have been dealing with, can we give them a particular timeline for when they can hope to be clinically assessed, if that still has to happen, but more importantly to have access to this drug in the same way it is available, for example, to our cousins in the North of Ireland under the National Institute for Health and Care Excellence, NICE, protocols? Will the drug be available under the same conditions as it is available in the UK under the NHS?

Sentiment score: 0.13