Transcripts for BBCNEWS Bittersweet Medicine 20240604 00:37:

BBCNEWS Bittersweet Medicine June 4, 2024 00:37:00

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, a bitter pill to swallow because nala can't be helped. so, you know, we're extremely grateful, in one sense,

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