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Young girl survives cancer thanks to little sister s lifesaving donation: A perfect match

A 10-year-old girl in the U.K. who had acute lymphoblastic leukemia is in remission thanks to her little sister, who was the perfect match for a bone marrow donation.

Mellbevágó: Az életéért küzd egy 4 éves kislány, miután kizuhant egy mozgó autóból

Mellbevágó: Az életéért küzd egy 4 éves kislány, miután kizuhant egy mozgó autóból
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Transcripts for BBCNEWS BBC News 20210811 12:51:00

it s a game changer. it is absolutely changed the face of sma. this is when he was seven weeks old in hospital. he had just been resuscitated by his dad. later diagnosed with severe spinal muscular atrophy. the vectors deliver new working copies of sma genes to the motor neuron cells. this new, one off drug contains a replica of the missing gene, halting the progression of the disease, helping babies breathe without a ventilator and even crawl and walk. he shouldn t really feel anything. he s got a cannula in, which is a little bit of plastic into his vein. and the drug just goes through that over an hour. injune, five month old arthur became the first baby to receive this revolutionary treatment on the nhs. now edward is one of a handful of babies being treated for the first time at the children s

Transcripts for BBCNEWS BBC News 20210811 13:50:00

through that over an hour. injune, five month old arthur became the first baby to receive this revolutionary treatment on the nhs. now edward is one of a handful of babies being treated for the first time at the children s hospital in sheffield. sma itself is actually the most common genetic condition that causes infant deaths and severe disability worldwide. about 90% of children with sma type one would die by age two. so, this treatment would drastically change the disease progression and outcome of each patient. you don t know what s around the corner. there s only five years worth of data. so, from what i ve seen from other children, theyjust get stronger. so, the world really is his oyster. thank you, guys. edward has no idea he has just received the most expensive drug in the world, or how his life will now be transformed. but his parents feel

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