3 minute read
Apr. 19, 2022
Branaplam: An Oral Brain-Penetrant Splicing Modulator to Reduce mHTT
branaplam
oral, CNS-penetrant splicing modulator w/ HTT activity in Ph. II for Huntington's (prev. SMA) further characterization of SMA candidate Nat. Commun., March 3, 2022 Novartis, Cambridge, MA
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