Miracle of £1.79m wonder drug baby
Single-dose treatment leads to first birthday joy
SHE was born with one of the rarest and most debilitating conditions known to medicine.
But now baby Isabella Winfield has beaten the odds and celebrated her first birthday thanks to a wonder drug that cost £1.79million for a single dose.
Isabella was only 20 days old when she was given Zolgensma, a gene therapy drug for spinal muscular atrophy (SMA).
Her condition was so severe that without the drug she would have been on constant life support.
But her parents say the the drug has allowed her to develop the ‘mobility of a child without SMA’.
Her mother, Margaret Paluszynska, 36, said: ‘Isabella is doing great. She has already gone above the average on the statistics her doctors have for children that have had Zolgensma treatment. She is a clever little girl who is physically better and stronger at doing things now. She is exactly where the doctors would have wanted her to be.
‘She has gained a lot of mobility in her upper body and for the first time she can lift her legs independently if she is lying on her back.
‘At Christmas, she could only do this in the bath where the water bore her weight. She can hold her head up for longer now than she could a few months ago and turn it quite quickly.
‘Before, when she would lift her head, it would flop back on her chest. And she is able to sit in a normal position on the floor now. Isabella has more of the mobility of a child without SMA at six months of age. None of this mobility would have been possible without modern medicine.’
Isabella, from Archiestown, Moray, was born on April 30 last year with the most severe form of the muscle-weakening illness – and received the single-dose drug just a week after diagnosis.
She was the youngest child in the UK to be given the drug and among the youngest in the world. Five days later, fivemonth-old Arthur Morgan became the first baby in England to receive it.
In June, it was revealed how doctors and Isabella’s family hoped it would transform her life. Her Type 1 SMA occurs in only three cases each year in Scotland and causes a range of life-shortening health issues, including breathing difficulties. Isabella’s father, Richard Winfield, 37, said: ‘If Isabella hadn’t had the Zolgensma when she did, at this stage of her life she would almost certainly have been on constant ventilation and feeding through a tube.
‘The drug means she hasn’t degraded to a point where we need all these extra help systems.’
Miss Paluszynska added: ‘Isabella would have had no improvement from a developmental point of view without this treatment. She would have been on life support.
‘There is no guarantee she would have even seen this birthday. This is such a happy day.’
‘This is such a happy day’