Table 1.

List of histone deacetylase (HDAC) inhibitors used in spinal muscular atrophy models to date. Included are inhibitors that have been shown to increase survival of motor neuron (SMN) RNA and/or protein levels in vitro and/or in vivo, as well as their effective dosage range and HDAC targets [6, 8–15, 19, 20]

Inhibitor name	Chemical class	Effective dose range	HDAC targets	Effect on SMN	Disadvantages
Phenylbutyrate	Short-chain fatty acid	mM	N/A	Yes
Sodium butyrate	Short-chain fatty acid	mM	Class I, IIa	Yes	Short half-life
Valproic acid	Short-chain fatty acid	mM	Class I, IIa	Yes	Hepatotoxic, teratogen
m-Carboxycinnamic acid bishydroxamide	Hydroxamate	µM	N/A	Yes	High toxicity
Suberic bishydroxamic acid	Hydroxamate	µM	N/A	Yes	High toxicity
Suberoylanilide hydroxamic acid	Hydroxamate	µM	Class I, II	Yes
Trichostatin A	Hydroxamate	nM	Class I, II	Yes
LBH589	Hydroxamate	nM	N/A	Yes
M344	Benzamide	µM	N/A	Yes	High toxicity
MS-275	Benzamide	µM	HDAC 1, 2, 3, 9	No

NA not available