06-02-2021, 04:59 PMDan B
News about drug Zolgensma used to treat SMA
The news media in the U.K. have reported that the first child there was treated with a new drug called Zolgensma, which is a gene-editing therapy that corrects the faulty gene causing Spinal Muscular Atrophy or SMA.
Obviously this is not the same as SBMA or Kennedy's Disease, but still, the notion that gene editing is being used to cure a neurological disorder is very encouraging.
Here's the article. https://www.pmlive.com/pharma_...py_zolgensma_1371002