BBCNEWS BBC News at One June 1, 2021 12:04:00
Cells from gradually dying. the now healthy motor neuron cells start producing the missing smn protein, which is vital for muscle function. evelina children's hospital in london is one of a handful of centres in the uk which will offer this groundbreaking 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. edward is eight months old. his parents hope he will be eligible for zolgensma, but they know the longer the treatment is delayed, the less effective it...