Table 3.

Preclinical small molecules in development for SMA.

#	Structure	Mode of Action	MWa	cLogPb	PSAc	HBAd	HBDe	Reff
25		Unknown (Post-transcriptional)	282	3.5	51	4	1	99, 103
28		Unknown (Transcriptional)	410	3.2	57	5	1	99
31		Unknown (Post-transcriptional)	360	2.7	71	7	1	105
34		Glycogen synthase kinase 3-β inhibitor	422	3.9	59	5	1	108
35		Unknown (Transcriptional)	605	1.3	194	12	7	109
36		Read-through inducing	483	−7.9	288	15	16	111

^aMW, molecular weight;

^bcLogP, calculated LogP;

^cPSA, polar surface area;

^dHBA, hydrogen-bond acceptor;

^eHBD, hydrogen-bond donor;

^fRef, reference number