BABY ARTHUR: 5 month old is one of the first patients ever to receive potentially life saving treatment in England
Five month old baby Arthur is one of the first patients in England to receive life saving treatment for spinal muscular atrophy.
A five-month-old baby has become the first patient in England to be treated with a potentially life-saving drug on the NHS that can prolong the lives of children with spinal muscular atrophy.
Arthur Morgan, who was diagnosed with the condition in May, received the one-off gene therapy, known as Zolgensma, at Evelina London Children’s Hospital on May 25.
Spinal muscular atrophy is a rare condition. Around 40 babies with the condition are born each year. If they are left to go untreated, they rarely live past two years old.
The condition will often lead to weakness of voluntary muscles, with arm, leg and respiratory muscles being affected first. Other issues may include poor head control, difficulties swallowing and scoliosis.
Zolgensma, a gene therapy manufactured by Novartis Gene Therapies, comes with a price tag of £1.795 million. It is understood that the NHS were able to obtain the therapy at a discount, meaning that dozens of infants will now be able to receive treatment for the condition each year.
Dr Elizabeth Wraige, a consultant paediatric neurologist at the hospital where Arthur was treated, spoke to the BBC about the treatment, and explained it can really alter the outlook for these children.
"Twenty years ago a baby born with SMA would have a very shortened life expectancy," she told the broadcaster.
"Now, we have the potential to enable children to acquire motor skills such as rolling and sitting and even standing-walking, that would have been impossible without treatment for SMA."
Video from PA, NHS / Evelina London Children's Hospital
Watch the channel on TV