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The FDA just approved zilganersen (Zanvastro), the first-ever treatment for Alexander disease, a progressive and often fatal brain disorder affecting fewer than 1 in 1 million people. This antisense oligonucleotide directly targets the buildup of a protein called GFAP, which damages brain cells. For patients and families who've had only supportive care, this is a true landmark.
This approval is a powerful signal of the precision medicine era. It's not a...
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