Table 1.

Small molecules that were repurposed for the treatment of SMA.

Name	Structure	Mode of Action	MWa	cLogPb	PSAc	HBAd	HBDe	Ref
Sodium butyrate (1)		Weak class I HDAC inhibitor	110	0.6	26	2	1	39
Sodium phenylbutyrate (2)		Weak class I HDAC inhibitor	186	2.0	26	2	1	40,45–49
Valproic Acid (3)		Weak class I HDAC inhibitor	144	2.8	37	2	1	41,50–54
Suberoylanilide hydroxamic acid (4)		Potent HDAC class I and II	264	1.0	78	5	3	42,43
Trichostatin A (5)		Potent HDAC class I and IIb	302	1.9	70	5	2	44,55,56
Riluzole (6)		Neuro-protective	234	3.2	48	4	2	57–62
Hydroxyurea (7)		Increases Nitric Oxide	76	−1.8	75	4	4	63–66
Ceftriaxone (8)		Unknown	554	0.02	209	18	5	67–69
Albuterol (9)		β2 Adrenergic Receptor Agonist	239	0.06	73	4	4	70–74
Aclarubicin (10)		Antibiotic	812	3.4	217	16	4	75, 76

^aMW, molecular weight;

^bcLogP, calculated LogP;

^cPSA, polar surface area;

^dHBA, hydrogen-bond acceptor;

^eHBD, hydrogen-bond donor.