going to be a game changerfor us and give arthur the best possible life he could have. around one in every 10,000 babies is born with spinal muscular atrophy. what is remarkable is that this one off treatment may permanently stop any further decline in arthur s muscle function. so how does zolgensma work? it contains a healthy copy of a missing orfaulty gene called smn 1. this is inserted into a harmless virus. in the body, the virus delivers the replacement gene into the nucleus of motor neuron cells. this is essential to prevent those cells from gradually dying. the now healthy motor neuron cells start producing the missing smn protein, which is vital for muscle function. how s arthur? evelina children s hospital in london is one of a handful of centres in the uk which will offer this ground breaking gene therapy.
copy of a missing, orfaulty, gene called smni. this is inserted into a harmless virus. in the body, the virus delivers the replacement gene into the nucleus of motor neuron cells. this is essential to prevent those cells from gradually dying. the now healthy motor neuron cells start producing the missing smni protein, which is vital for muscle function. how s arthur? evelina london children s hospital is one of a handful of centres in the uk which will offer this ground breaking gene therapy. i think it has the potential to make a very significant difference, to enable children to acquire motor skills such as rolling and sitting, that would have been impossible without treatment for sma. she was really floppy. she couldn t sit, - she couldn t hold toys. she couldn t do anything. tora received zolgensma in the us nearly two years ago.
so, this is the world s most expensive medicine, with a list price of almost £1.8 million. the nhs has negotiated a confidential discount, which will mean that dozens of affected infants can be treated every year. zolgensma s benefits last at least five years and might even be permanent. that partly explains its sky high cost. but with other gene therapies on the horizon, that raises the issue of affordability. so, how does zolgensma work? it contains a healthy copy of a missing, orfaulty, gene called smn1. this is inserted into a harmless virus. in the body, the virus delivers the replacement gene into the nucleus of motor neuron cells. this is essential to prevent those cells from gradually dying. the now healthy motor neuron cells start producing the missing smn1 protein,
should prevent further loss of nerve cells. it s been massive, massive ups and downs, but being able to now have the treatment is just going to be a game changerfor us and give arthur the best possible life he could have. around one in every 10,000 babies is born with spinal muscular atrophy. what is remarkable is that this one off treatment may permanently stop any further decline in arthur s muscle function. so how does zolgensma work? it contains a healthy copy of a missing orfaulty gene called smn i. this is inserted into a harmless virus. in the body, the virus delivers the replacement gene into the nucleus of motor neuron cells. this is essential to prevent those cells from gradually dying. the now healthy motor neuron cells start producing the missing smn protein,