“Look into my son’s eyes and tell him his life is not worth saving” was the heart-breaking message to Health Minister, Simon Harris, by a Youghal mother. Rebecca Bulman was among the parents of 25 other children with a rare muscle-wasting condition, who are campaigning for access to the drug Spiranza.
Spinal Muscular Atrophy (SMA) is a genetic fatal disease in infants where one of two muscle building genes are missing. A child with the condition is unable to sit or speak. SMA has four types. Children with type one rarely live beyond 2 years of age, while those like 5 year old Harry Bulman with type two has a life expectancy of his early 20s.
Speaking at a highly emotional cross party meeting in the Dáil Rebecca Bulman told TD’s it was “heart-breaking” that her 5 year old son is being denied the life changing drug. Young Harry is completely dependent, “he needs to be lifted out of bed to go upstairs to the toilet numerous times a day. He has to be turned at least seven times a day in bed because he can’t belch. He is on extra drinks to maintain weight because his chewing is getting slower. He’s on loads of machines if he gets sick to help the muscles in his chest” said Ms Bulman.
Spiranza is a treatment that can help the condition by putting extra proteins into the one remaining muscle building gene, helping maintain the muscle that is there and, in some cases, actually rebuilding muscle. Despite being available in 25 other European countries, children with SMA cannot access the drug through the HSE. The Department of Pharmaeconomics has found that a one year supply of the drug would cost over €640,000 per child, and each subsequent year that would drop to €341,000 per patient.
Marking World Rare Disease Day, Sinn Fein TD Pat Buckley said “Pharmaceutical Companies should not be holding children to ransom. These medicines should be based on medical need, not medical means.”
“If the UK exit Europe later this month, Ireland and Estonia will be the only countries in Europe not supplying the drug. Turkey, who is five times less off than Ireland, can broker a deal” said the Cork East Deputy.
The HSE was asked for comment, but failed to do so prior to going to print. Over 6,600 people have been treated with Spinraza in more than 40 countries.