cells from gradually dying. the now healthy motor neuron cells start producing the missing smn protein, which is vital for muscle function. evelina children s hospital in london is one of a handful of centres in the uk which will offer this groundbreaking 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. edward is eight months old. his parents hope he will be eligible for zolgensma, but they know the longer the treatment is delayed, the less effective it is. for his quality of life and how long his life could be, zolgensma isjust a game changer. and we re just kind of patiently waiting, we ve been waiting since last november, for zolgensma. some other infants treated with zolgensma have seen
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 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?
possible life he could have. around one in every 10,000 babies is born with spinal muscular atrophy. what is remarkable is 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 or faulty 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 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
around one in every 10,000 babies is born with spinal muscular atrophy. what is remarkable is 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 or faulty 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 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.
permanently stop any further decline in arthur s muscle function. so, how does zolgensma work? it contains a healthy copy of a missing or faulty 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 smn protein, which is vital for muscle function. evelina children s hospital in london is one of a handful of centres in the uk which will offer this groundbreaking 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. edward is eight months old.