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Life limiting disease. so how does a single faulty gene cause so much damage in children with mld? so there s mistakes in a gene, which means the body can t make an enzyme. the job of that enzyme is to break down a really important chemical for how our brain functions and works. and so, it gradually builds up and builds up in the brain until this catastrophic point, usually by the age of two, where children start to lose, as i said, all of those abilities. but now, there s a treatment for children with mld a form of gene therapy called libmeldy. it s a medicine which can help one of the shaws daughters, but not both. so when they told us that there was treatment available for teddi, it was, kind of, like, a bitter pill to swallow ....
I ve been looking after children with mld for 17 years now, and watching many children pass away because of this untreatable, incurable, and very life limiting disease. so how does a single faulty gene cause so much damage in children with mld? so there s mistakes in a gene, which means the body can t make an enzyme. the job of that enzyme is to break down a really important chemical for how our brain functions and works. and so, it gradually builds up and builds up in the brain until this catastrophic point, usually by the age of two, where children start to lose, as i said, all of those abilities. but now, there s a treatment for children with mld a form of gene therapy called libmeldy. it s a medicine which can help one of the shaws daughters, but not both. so when they told us that there was treatment available for teddi, it was, kind of, like, ....
Can t make an enzyme. the job of that enzyme is to break down a really important chemical for how our brain functions and works. and so, it gradually builds up and builds up in the brain until this catastrophic point, usually by the age of two, where children start to lose, as i said, all of those abilities. but now, there s a treatment for children with mld a form of gene therapy called libmeldy. it s a medicine which can help one of the shaws daughters, but not both. so when they told us that there was treatment available for teddi, it was, kind of, like, a bitter pill to swallow because nala can t be helped. so, you know, we re extremely grateful in one sense, and really sad on the other side, so. i can t really imagine ....