A MUM has said it has been hard seeing her babies suffering a rare life limiting condition but she has hope from the treatments they are receiving, including new pioneering therapy.
Justine Smith's sons Rocky and Bentley both have the most severe form of spinal muscular atrophy. Doctors often give babies with the condition just 24 months to live from birth.
Babies born with Type 1 spinal muscular atrophy (SMA) - the most common form - experience progressive muscle weakness, loss of movement and difficulty breathing.
Until two years ago there were no treatment options for children with the condition, which is the leading genetic cause of death for youngsters.
But research found that a single treatment of the gene therapy Zolgensma has helped babies with SMA to sit, crawl and walk, and also prevented them from having to be put on a ventilator.
And now five month old Bentley is one of the first to receive the treatment, which has a price of £1.79 million per dose and was made available on the NHS after the health service struck a deal with manufacturers earlier this year.
Mrs Smith, 26, said: "This can expand life expectancy.
"It can work wonders, it is fantastic. He would never be able to sit up, move, walk, eat and would need help with breathing.
BABY: Bentley
"But because Bentley has had this treatment he will not need or require feeding or bipap which is the machine which helps Rockey to breathe, and Bentley might be able to walk."
Sadly Rocky, now 22 months old, is too old for the gene therapy, but is responding well to another form of treatment for SMA, Spinraza, which works by helping the body to produce more functional SMN protein that people with SMA are lacking.
Mrs Smith said he had been having that treatment for four months.
"He had been in and out of hospital in intensive care with the common cold and the flu," she said.
MUM: Justine Smith, pictured with Rocky in 2020
TREATMENT: Rocky
"His heart, his windpipe are all pushed to the right of his body, we are not sure if he will have to be operated on.
"That treatment is working for him."
Mrs Smith said it was heart-wrenching to have one child going through it, but having two had been especially tough.
"It is such a slim chance, and they have the severe type, it is really hard," she said.
"I was talking to other parents of children with SMA - Rocky is one of the worst, he is in and out of hospital all the time."
The Upton-upon-Severn resident said she was now wanting to spread the word that Zolgensma was on the NHS so other families could consider it as an option.
And she is also supporting a campaign for screenings for SMA during pregnancy as, if treatment is delivered at birth, children have the best chance of long and healthy lives.
"Another mum told me doctors told her they couldn't have found out for her when she was pregnant," Mrs Smith said.
"But I did, I was pregnant when I found out. There needs to be more screenings for SMA during pregnancy."
The petition can be found at petition.parliament.uk/petitions and searching for 'Fund newborn screening for spinal muscular atrophy immediately'.
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