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. Author manuscript; available in PMC: 2015 Mar 11.
Published in final edited form as: Future Med Chem. 2014 Jun;6(9):1081–1099. doi: 10.4155/fmc.14.63

Figure 2. Select small compounds that have been investigated for spinal muscular atrophy therapy.

Figure 2

Select HDAC inhibitors that have been tested in mouse models of spinal muscular atrophy (SMA) and in the case of valproic acid in several clinical trials in SMA patients [58]. Rho kinase inhibitors [59,60] and the read-through compound TC007 [56] have also been tested in mouse models of SMA. Pseudocantharidin C modulates the activity of PP2A to increase SMN2 exon 7 inclusion [61]. The neuroprotective compound olesoxime is currently being tested in a Phase II clinical trial of type II and III SMA patients [62].