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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 a...

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....

Without treatment, those affected rarely live beyond the age of two. arthur is unable to lift his head, and struggles to move his arms and legs, but this single infusion 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 this one—off treatment may permanently stop any further decline in arthur's muscle function. so how does zolge...

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 shorten...

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, 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, t...