A new drug approval for the vanguard of RNA-targeted small molecules
Dark Matter Blog
AUGUST 27, 2020
A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. On the strength of those findings, nusinersen was approved by the FDA at the end of 2016 for the treatment of SMA , marketed under the commercial brand Spinraza.
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