Novartis and gene therapy to treat Rett Syndrome.
Novartis, a multinational biotechnology company, issued a statement in August 2020 to confirm full commitment to their gene therapy program for Rett Syndrome. This was followed by a press release from the company in March 2021 which reported recent results of gene therapy to treat patients with spinal muscular atrophy (SMA). These results are of enormous interest to those of us who are related in some way to girls with Rett Syndrome and have important ramifications. In this short article, I have attempted to explain why this is so.
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