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BBCNEWS BBC News at Six June 1, 2021 17:14:00

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, 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. ....

Couldnt Fight , Replacement Gene , Motor Neuron , Smn1 Protein , Muscle Function , Evelina London Childrens Hospital , Gene Therapy , Motor Skills , Spinal Muscular Atrophy , She Couldn T , She Couldnt Hold Toys , She Couldnt Sit ,

BBCNEWS BBC News June 1, 2021 14:19:00

It s been 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 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. evelina children s hospital in london is one of a handful ....

Game Changerfor Us , Ups And Downs , Spinal Muscular Atrophy , S Muscle Function , Smn Protein , Muscle Function , Replacement Gene , Motor Neuron Cells , Motor Neuron , Evelina Children S , In London ,

BBCNEWS BBC News at Ten June 1, 2021 21:18:00

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, which is vital for muscle function. how s arthur? evelina london children s hospital ....

Gene Therapies , Couldnt Fight , Replacement Gene , Motor Neuron , Smn1 Protein , Muscle Function , Evelina London Childrens Hospital ,

BBCNEWS BBC News June 1, 2021 10:04:00

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. ....

Gene Therapy Treatment , Arthur Morgan , Game Changerfor , Missing Orfaulty , S Muscle Function , Replacement Gene , Motor Neuron Cells , Motor Neuron , Smn Protein , Muscle Function , In London , Evelina Children S ,

BBCNEWS Outside Source June 1, 2021 18:24:00

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. ....

Replacement Gene , Motor Neuron , Evelina London Childrens Hospital , Missing Smni Protein , Muscle Function , Gene Therapy , Motor Skills , Spinal Muscular Atrophy , She Couldn T , She Couldnt Sit , She Couldnt Hold Toys ,