A Rathcoole family are calling on the Health Minister to make a drug available that could drastically improve their son’s quality of life.
Fiona and Paul Bailey’s son Sam (8) has Spinal Muscular Atrophy (SMA), a degenerative neuromuscular condition, characterised by generalised weakness of all the muscles in the body.
SMA is a debilitating, life-threatening and rare disease and is the leading genetic cause of death in infants.
Previously, there was no treatment or cure for SMA but a new drug, Spinraza, has been licensed in 20 countries across Europe.
The drug has the potential to seriously improve the lives of those with SMA.
The family have started a change.org petition calling on Minister Simon Harris to make the drug available in Ireland.
Fiona and Paul said: “Sam is in a powered wheelchair. He is peg fed and so does not eat or drink orally.
“Extensive chest physio and physio on all his limbs to keep him supple and free from pain is part of his everyday routine.
“He has already endured so much in his short life, he’s had long hospital stays, sometimes for weeks and months at a time, largely because of chest infections etc. Life is very difficult for our darling Sam.”
Despite his struggles, the family say Sam is the “brightest, sweetest, kindest boy you could ever meet”.
“He has two beautiful sisters, Kayla (6) and Sarah (2). He loves school and all his friends at Scoil Chronain,” they said.
“He loves football and he is definitely West Ham United’s biggest fan (he still thinks they can win the Prem having lost their first four games!).”
The couple said Spinraza’s availability has created hope for people living with SMA the first time.
“It has been described as a ‘miracle drug’ because it changes children’s lives,” they said.
“Spinraza is making a momentous difference in the lives of those who, previously, had no other options and who, for some, faced death.”
They said they started the petition to call on Minister Harris to approve funding for the drug in Ireland for Sam and the 25 other children who have SMA.
“Time is not a luxury for Sam and the other 25 children in Ireland waiting on this drug. SMA is a degenerative condition, we need this life saving drug today.”
Dublin Gazette contacted the HSE, but had not received a reply by the time of going to press.