How to pronounce“zolgensma”in English
IPA/zɒldʒɛnsmæ/
Syllableszol·gen·sma
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10:51
This disease, prior to gene therapy, was always fatal at a very young age. Children would die usually around two or three years old.
With Zolgensma though, if children are treated very early, in the first few weeks of life, say, the gene therapy can restore the function of that gene.
It can, with a one-time treatment, completely cure the disease.
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