An appeal has been launched for the HSE to give approval for a 'miracle drug' that could save the lives of very small children who have a rare muscular condition.
Two children in Co Cork and Co Kerry are understood to be the only suitable possible recepients for the drug, Zolgensma, which is used to treat spinal muscular atrophy, a genetic condition leading to loss of movement. The treatment has been hailed as a 'miracle drug' that can lead to vastly improved quality of life and lengthened lifespan.
The drug has already been rejected by health authorities in Ireland on the basis of cost, but negotiations are taking place with the makers of Zolgensma to reduce the price. It costs around €2m for the one-off treatment and can only be administered to children typically under the age of two, based on their weight.
Lorraine Mynard, from Dunmanway, fears that any approval for the drug will be too late for her daughter, Kate, who is 17 months old.
"She is running out of time in that respect," Lorraine says. "I have been looking on YouTube at the stories of children who have got the treatment in places like the US and it makes a huge difference to their lives.
"Kate was recently in hospital for two months, with respiratory issues, in Temple Street in Dublin, and in Cork," Lorraine says. "She can't take anything orally, at the moment, and Zolgensma would improve her swallowing muscles greatly. It's pure torture and very frustrating that this isn't here already. I've been in contact with every TD and the HSE trying to get things moving."
Theo Whelan, who was born last April, in Tralee General Hospital, and lives with his parents, Shane and Stephanie, in Killorglin, Co Kerry, is the other possible recipient of Zolgensma. Theo's father, Shane, says that the treatment would hugely improve Theo's quality of life, particularly in relation to feeding muscles and his respiratory system.
“We are appealing to the HSE and the Government to come to an agreement on the price," Shane says.
"Even though it is €2m, it should be put in the context of the price of the drugs that are currently being administered for the condition and the fact that this is a once-off treatment. Theo has been doing very well; he did have an episode where he nearly choked, which is related to his condition, but we have been able to manage."
The condition has necessitated extended stays in hospital and constant travelling and relocation for both sets of parents. "They are doing an amazing job in Temple Street," Shane says. "But, as parents, you need to be trained up about so many things in order to manage this."
Last May, five-month-old Arthur Morgan became the first child in the UK to receive the drug, after the NHS struck a deal with the manufacturers, Novartis, on the price.
A spokesperson for the HSE says that the Corporate Pharmaceutical Unit is engaging with colleagues in the Netherlands and Belgium in a joint negotiation with the drug manufacturer.