Baby Arthur is just five months old. He has no way of knowing the treatment he is receiving in his right arm is the world’s most costly medicine.
His devastating progressive disorder causes loss of muscle control.
But he has become one of the first patients to be treated on the NHS with Zolgensma, a gene therapy with a list price of £1.795m.
Around 40 children are born with the most severe form of Spinal Muscular Atrophy (SMA) every year.
Untreated, they rarely live beyond two.
The NHS has negotiated a confidential discount which means dozens of infants can be treated each year with Zolgensma, which is manufactured by Novartis Gene Therapies.
Arthur, from south-east London, finds it difficult to move his arms and legs and cannot lift his head, so some permanent damage has already been done.
The hope is the one-off treatment will stabilise his condition and prevent further deterioration.
His father, Reece Morgan, says there have been many ups and downs.
“Our hope is that he can have the best possible life in terms of his movement,” he says. “We don’t know, but we’re just going to try as best as we can to give him everything he possibly needs.”
