‘Miracle baby’ with rare Spinal Muscular Atrophy given less than 2 years to live as he misses out on NHS funding by 5 weeks
The parents of an eight-month-old baby boy have received the devastating news that their son may not reach his second birthday because of his extremely rare genetic disease, Spinal Muscular Atrophy (SMA Type 1).
Tara and Mo Albaali, the parents of Zakariya - known as Zak - hope to raise £1.9 million to pay for life-changing US gene therapy, Zolgensma, , which was approved by the NHS in March to treat babies with the fatal disorder.
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Around 80 babies and young children with type 1 SMA - the most severe form of the illness - could benefit from the treatment each year in England, say experts.
However, Zak’s parents claim on his GoFundMe page that they were told that the NHS only funds the treatment for babies aged up to six months.
“My consultant said if she had the choice, she would give me Zolgensma tomorrow as it will provide my body with the missing gene, which is a one-off IV infusion,” said a message written from the perspective of Zak on his page.
His mother Tara pleads with the public on the page to “allow us the most amount of time with our son”, and donate as much as possible as Zak could die in the next 14 months if he does not receive treatment.
‘Zak has missed all his milestones’
SMA affects the central nervous system and voluntary skeletal muscle movement. Consequently, affecting many basic motor functions, some of which are walking, leg and hand movements and feeding.
As the illness progresses, patients suffer from loss of the ability to swallow, breathe and the likelihood is that they will eventually die from the degenerative condition.
Zak, who was born in October 2020,received his diagnosis on 18 May, since then he has already lost the ability to use his legs and is slowly losing power in his arms.
Tara and Mo said of his condition: “Zakariya has missed all his milestones, he has never rolled over, sat unassisted, he has a little strength in his head, but it is very tiring for him to hold for long, he has completely lost the ability to lift or move his legs - only his feet move - and his arms are considerably weaker.
“There is still a huge risk of respiratory failure, ultimately leading to death.”
‘Give our baby boy a second chance at life’
Zak’s parents say that he is in urgent need of the drug Zolgensma, which is given in a one-off IV infusion and replaces the gene he lacks.
It is not known whether his legs could be rehabilitated if he was to receive the treatment, but he would be granted a longer life expectancy and greater quality of life.
The money donated would go towards paying for the drug - the world’s most expensive pharmaceutical treatment - and his aftercare.
His parents told donors in their pledge: “When we reach our target, we will have several hospitals to choose from either within the UK or in the US at Boston Children’s hospital.
“The current US quote stands between $2.2 - $2.9 million which does not include travel expenses, accommodation and aftercare costs.”
Tara and Mo said it would “give our baby boy a second chance at life.”
The drug has proven to work effectively and some children have even gone on to walk, according to reports.
The couple had struggled for five years to have a child before giving up on their hopes after five years of failed IVF attempts.
It was two months after moving to Dubai to get “far away from [their] struggles” that they found out they had naturally conceived Zak.
They then moved back to the UK when he was born last year.
Tara and Mo said they will do whatever it takes to save their only child, adding: “we do not want to be on this earth unless we have done the utmost for Zaki.”