in the world, obviously. what s incredible is that this treatment may permanently stop any further treatment may permanently stop any furthe . ,. . ., 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 smn1 protein. this has opened up a door for other genetic diseases, cystic fibrosis, and all types of other rare diseases. matthew hill, bbc points west. some other infants have seen their condition notjust stabilise, but in proof, and week later, riley s mum is very optimistic.
has negotiated a prevent further cell loss. the nhs has negotiated a confidential- has negotiated a confidential discount which will enable dozens of affected infants to be treated each year but doctors still needed to be sure it would help riley. he year but doctors still needed to be sure it would help riley. year but doctors still needed to be sure it would help riley. he had to be able to come sure it would help riley. he had to be able to come off sure it would help riley. he had to be able to come off the sure it would help riley. he had to be able to come off the ventilatorl be able to come off the ventilator for eight hours a day at least otherwise there is no quality of life. he has been booming with his movement and breathing so they decided to have a big meeting about it and he was approved for it which was the best feeling in the world, honestly. was the best feeling in the world, honestl . ., ~:: :: :: , , was the best feeling in the world, honestl. ., ~::::::
virus. this delivers the replacement gene into the nucleus of motor neuron cells. this is essential to prevent themselves gradually dying. the now healthy cells start producing the missing protein. this is the opened producing the missing protein. try 3 is the opened up to wear for other genetic diseases, cystic fibrosis and all sorts of other diseases. some other infants treated with this drug have seen their condition not just stabilise but improve, and a week after riley s infusion, his mum is very optimistic. is very optimistic. they say improvement is very optimistic. they say improvement starts - is very optimistic. they say improvement starts to - is very optimistic. they say i improvement starts to happen is very optimistic. they say - improvement starts to happen after 3-5 improvement starts to happen after 3 5 months, but i m already starting to see some slight improvements already, so ijust can t wait to see some slight improvements already, so i just can t wait to see
quality of life there, so we managed to do that. he is been improving with his movement and his breathing, so they decided to have a big meeting about it and he got approved for it, which was the best feeling in the world, obviously. so how does the drug were? so how does the drug 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 smn1 protein. this has opened up a door for other genetic diseases, cystic fibrosis, and all types of other rare diseases. matthew hale, bbc points west. matthew hill, bbc points west. the world health organisation has urged extreme caution
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 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