the list price of zolgensma is almost £1.8 million. negotiated a confidential discount which will enable dozens of affected infants to be treated each year. five—month—old arthur is one of the first to benefit. he has spinal muscular atrophy, sma, a genetic condition which causes progressive muscle weakness. 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 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 t

but it says amount build, 6,193,647.16. >> the drug alone zolgensma cost $2 million.nd the drug says it only costs about half of what a family would otherwise spend on treatments over a decade. as far as what families actually pay, the company says nearly all its u.s. patients are covered by insurance. when did you start seeing those tantalizing signs that wow, this thing is working? >> by day three, she actually lifted her head up. >> oh my goodness! >> okay, so why are you crying? >> well, today reese has started sitting. unassisted. let her go. and this is just -- it's a really big deal. just really grateful. >> i've seen video where she turns over from her side on to her back for the first time. and i hear you guys rooting her on and celebrating. >> whoa, reesy! >> any time she does anything, it's a celebration. i feel like we want to throw a party. >> they did get to throw reese a party, her first birthday. pandemic-style. ♪ happy birthday dear reesie ♪ >> just the three of them. ♪ happy birthday to you. >> she has made some wonderful gains. she is lifting her ar

zolgensma's benefits last at least five years and might even be permanent.rapies 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 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 sa

this particular drug, zolgensma. — screening. this particular drug, zolgensma, why _ screening.icular drug, zolgensma, why is it _ screening. this particular drug, zolgensma, why is it so - screening. this particular drug, - zolgensma, why is it so expensive? very good question, at nearly £1.8 million. it is partly to do with the costs of development and it is also partly to do... because all new drugs are expensive, when there are a limited number of patients who will receive it, it's a very complex decision in terms of pricing. but knights and the scottish medicines consortium, both considered the balance between the benefits and the cost, and both of them said that zolgensma was cost—effective, because it is so transformative. —— nice. because you have to set it against the children who would need 24—hour support for the rest of their lives, or another costly treatment. but it is hugely expensive. but the nhs has made this confidential deal and it means around a0 children, babies, each year, will be treated to. around 40 children, babies, each year, will be treated to.- year,

so how does zolgensma work? it contains a healthy copy of a missing or faulty gene called smn1. 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. in the past few hours, a dedicated terminal for anyone arriving on a flight from any of the 43 nations on the uk government's �*red list�*, has opened at heathrow airport. it follows criticism that passeng

the list price of zolgensma is almost £1.8 million.e nhs has negotiated a confidential discount which will enable dozens of affected infants to be treated each year. five—month—old arthur is one of the first to benefit. he has spinal muscular atrophy, sma, a genetic condition which causes progressive muscle weakness. 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 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 smni. this is inserted into a harmless virus. in the body, the virus d

zolgensma's benefits last at least five years and might even be permanent.hat 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 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 basical

zolgensma's benefits last at least five years and might even be permanent.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 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 d

arthur morgan, who has spinal muscular atrophy, is being treated with zolgensma.n per patient it is the world's most expensive drug. the nhs has negotiated a confidential discount for the treatment, which can increase life expectancy by as much as a0 years. the development means that potentially dozens of infants could be treated by the nhs. around a0 children are born with the most severe form of the disorder every year. and the sma uk charity says newborns in britain should be screened for sma, as happens in other countries. the charity says early diagnosis and treatment is vital. 0ur medical editor, fergus walsh, explains. this is the world's most expensive medicine. the list price of zolgensma is almost £1.8 million, though the nhs has negotiated a confidential discount, which will enable dozens of affected infants to be treated each year. five—month—old arthur is one of the first to benefit. he has spinal muscular atrophy, sma, a genetic condition which causes progressive muscle weakness. without treatment, those affected rarely live beyond the age of two. ar

zolgensma's benefits last at least five years and might even be permanent.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. 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, i like, basic and simple for any other child. now we look forward with optimism that tomorrow something good will happen. the sooner babies with sma are treated, the better the outcomes. there's now pressure to add the condition to the newborn screening programme so that zolgensma can be given in

i feel like zolgensma is the carrot that has been _ zolgensma is the carrot that has been dangled inble. megan, great to talk to you. i am sure you would be - you. i am sure you would be interested in our next guest. take care of yourself and look after edward. thank you for having a beautiful baby. thank you very much. thank you. we're joined now by our medical editor, fergus walsh. you can see there the difference that this drug would potentially make. how significant is this development? it’s make. how significant is this development?— development? it's hugely significant. _ development? it's hugely significant. it _ development? it's hugely significant. it was - development? it's hugely significant. it was such i development? it's hugely significant. it was such a | development? it's hugely - significant. it was such a pleasure to be _ significant. it was such a pleasure to be going into an nhs hospital and reporting _ to be going into an nhs hospital and reporting firstly on something apart from cawood and secondly on such an innovation _ from cawood and secondly on such an inno

so, how does zolgensma work? it contains a healthy copy of a missing or faulty gene called smn1.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. amazing. a medical breakthrough. until recently, putting forward your own theories about ufos at a dinner party would leave you with polite nods and not—so—discrete eye—rolls, and chances are, the hosts wouldn't invite you back. but change is afoot here in america, where there is a growing bipartisan consensus that they are worthy of further study. any day now, the pentagon is due to deliver an unclassified report to congress, setting ou