Mom Ally Wallace and dad Jake Shaw learned that their daughters, Nala, 2, and Teddi, 11 months, have Metachromatic Leukodystrophy (MLD), a brain disorder that impacts their physical and mental development.
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.
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. let s hear now about a little girl called toya who has had this torah. gene therapy treatment. tora was able to have the treatment while her family were living in new york. herfamily are now back in the uk. her dad rajdeep patgiri joins us from south west london. it s it s extraordinary he is getting this treatment. i know you had to go through hoops for your daughter. tell us about her and how you