Julie Helen: Time is ticking for people waiting for drug approvals 

I often think about the campaigns we see for orphan drugs for rare conditions to be provided by the HSE to people who need them.

Everybody knows I have cerebral palsy, a lifelong condition. I was born with. It doesn’t technically impact my life expectancy, but in real terms, ageing is harder, so my condition doesn’t get worse, but it changes. I have to work very hard to make sure those changes don’t significantly impact the way I live my life. Sometimes, I have to accept that changes will be more permanent. I used to be able to walk into the shop with my two walking sticks, walk around, buy 12 to 15 items, and carry them out to the car before my legs would run out of energy. I cannot do that now. I would be at a much higher risk of falling, or I might just run out of steam halfway through. It is frustrating and exhausting. My voice remains strong, and it will stay that way until I am 90 or beyond. My arms are strong and getting stronger, and I can work on my leg strength by standing and walking every day and by losing weight.

There is no force like a ticking time bomb working against me. For children and young people with Duchenne Muscular Dystrophy and Friedrich’s ataxia, it is different. These conditions lead to weakened muscles and impaired coordination, respectively, over time, and once power is lost, it doesn’t really come back because the conditions keep progressing.

In recent weeks, the HSE sanctioned the provision of Givinostat, a life-changing drug for children, primarily boys, who have Duchenne Muscular Dystrophy. The condition affects around 100 boys in Ireland. The important thing to remember is that there will be little boys diagnosed in the future, and having the drug immediately will change their outlook drastically in a positive way. My heart goes out to families whose boys have already died due to the condition, or the boys who have so much progression of the disease that they may not be eligible to have it. Medicine didn’t move fast enough for them, and that is heartbreaking. All I can think is if it were my little boy, the absolute light of my world, how devastating it would be. I watched the campaign unfold, willing groups like “Join our Boys” and Una Ennis on; they succeeded, they have changed Ireland for the boys of today and of the future.

Advocacy is a hard and relentless road, and now the community who have Friedrich’s ataxia is still in a similar fight, begging for Skyclarys for about 200 people living with the debilitating and progressive condition. It robs people of their independence, of their voices, and ends in death. I could use prettier words, but sanitised language won’t properly convey the solidarity I feel with the people who had to use all their energy to sit in their wheelchairs outside the Dáil, wondering how much each of their lives is worth to our Government. I watch gorgeous people like Niamh Ní Hoireabhaird, Emily Felix, and Aoife Quinn speak openly and eloquently about their personal situations. I don’t know any of them personally, but as a fellow disabled woman, I applaud their grit and bravery, but I am so cross that they have to put their privacy and difficulties on the line to make the powers that be listen.

I am lending my voice in the best way I can because every single life matters, and time is ticking by.