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Whether or not a subset of younger non-ambulatory SMA subjects might benefit from VPA treatment early in the course of their disease over a longer treatment period remains an outstanding question
doi: 10.1016/j.cbi.2020.109093 600 ChongZZMaieseK
Pathak D, Shields LY, Mendelsohn BA, Haddad D, Lin W, Gerencser AA, et al
The mechanisms of human hotdog-fold thioesterase 2 (hTHEM2) substrate recognition and catalysis illuminated by a structure and function based analysis
P., Reilly, S
