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BBCNEWS BBC News June 4, 2024 12:54:00

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. ., ~:::::: ....

Cell Loss , Confidential Has , Muscle Function , Replacement Gene , Motor Neuron ,

BBCNEWS BBC News June 4, 2024 19:57:00

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 ....

Replacement Gene , Motor Neuron , Cystic Fibrosis , Christian Fraser , Keith Lucas ,

BBCNEWS BBC News June 4, 2024 20:50:00

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 ....

Replacement Gene , Smn1 Protein , Motor Neuron Cells , Cystic Fibrosis , Motor Neuron , Bbc News , Points West , Matthew Hill , World Health Organisation , Matthew Hale ,

MSNBC Way Too Early With Kasie Hunt June 4, 2024 09:49:00

We are going to deliver on them. do you have any concerns about senator joe manchin and the way he s approached this process? he s really in many ways a one-man senate that is going to determine the fate of all of this. there s a lot of stakeholders, senate, house, white house involved in these negotiations. it is going to be a multi-faceted process. my focus right now is ensuring the excellent infrastructure bill we just passed in the house, which is making historic generational investments in transit and fix fixing our infrastructure, ensuring clean water really become the nucleus of the senate version of the bill. there s a lot of people at the table right now, and i m confident we re going to deliver a good end product. we talk about the infrastructure packages on the two-track solution, but it s very important to underscore you are, in fact, doing so much in the bill that you just passed and in the other standard ways ....

Joe Manchin , Infrastructure Bill , White House , Georgew Bush White House , Infrastructure Packages , End Product ,

MSNBC Katy Tur Reports June 4, 2024 18:44:00

Of the hierarchy that they want to secure information, but the trump organization is not as sprawling as some people are imagining, but it is a tight knit business of employees and nucleus, and his children, and mr. weisselberg and a few have been the linchpins of that for a long time. so without him, it is inevitably going to make it much more difficult to if they are not getting what they want to get through. all right. josh and and adam, when those charges are filed tomorrow, we will bring you that news. thank you for your time. meanwhile, former president donald trump is going to be in ....

Trump Organization , Alan Weisselberg , Knit Business , Adam Kauffman , Donald Trump ,