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.
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. for her, it s been transformative. it saved her life. and basically, she now has a chance to enjoy her life and to do things, that are so, . like, basic and simple for any other child. now we look forward with great optimism that tomorrow, something good will happen.
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,
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. 20 years ago, a baby born with a severe form of sma would have a very shortened life expectancy. we can now offer treatment that can really alter the outlook for these children. some other infants treated with zolgensma have seen their condition notjust stabilise, but improve. arthur s parents say they know it s not a cure, and they re simply delighted that he s being treated. fergus walsh, bbc news. the british medical association has joined calls for the uk government to delay a decision on lifting all remaining lockdown restrictions which is due to take place in england on 21stjune. some government scientific advisers