SMN2-Stablizer-27 is a stablizer of survival of motor neuron 2 (SMN2), and a post-translationally stablizing SMN protein for the treatment of spinal muscular atrophy (SMA).

Risdiplam (RO7034067) (RG7916) is orally administered. Risdiplam is a centrally and peripherally distributed SMN2 pre-mRNA splicing modifier which increases survival motor neuron (SMN) protein levels[1].

SMN-C2 is a selective regulator of SMN2 gene splicing, a risdiplam analogue, a selective RNA-binding ligand that regulates pre-mRNA splicing and acts by binding to SMN2 pre-mRNA.SMN-C2 has the potential to be used in the study of spinal muscular atrophy (SMA).

SMN-C3 (MV8T2MCK57) is an orally active modulator of SMN2 splicing, and has the potential to treat spinal muscular atrophy (SMA).

Nusinersen, an antisense oligonucleotide drug, modifies the pre-messenger RNA (pre-mRNA) splicing of the SMN2 gene, thereby enhancing the production of the full-length Survival Motor Neuron (SMN) protein.

RG7800 hydrochloride is an orally active modulator of SMN2 splicing.

Branaplam is a highly potent, selective and orally active SMN2 splicing modulator. Branaplam increases the amount of functional SMN protein produced by the SMN2 gene through modifying its splicing.

Homocarbonyltopsentin is a small-molecule TSL2-binding compound, binds to pentaloop conformations of TSL2 and promotes a shift to triloop conformations that display enhanced SMN2 exon 7 (E7) splicing (EC50: 16 μM).

RG7800 hydrochloride is an orally active modulator of SMN2 splicing, exhibiting EC50 values of 23 nM for SMN2 splicing and 87 nM for SMN protein production. This compound holds promise as a therapeutic agent for spinal muscular atrophy.

Branaplam (LMI 070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.

RG7800 (RO6885247) has the potential for spinal muscular atrophy treatment. RG7800 is an SMN2 splicing modifier.

NVS-SM2 is an effective small molecule splicing enhancer of SMN2 with oral activity. NVS-SM2 showed increased exon 7 inclusion and upregulated SMN protein expression in SMA fibroblasts and SMN cells Δ7 5025 mouse myoblasts.

RG7800 hydrochloride is an orally active SMN2 splicing modulator that acts on SMN2 splicing (EC1.5x: 23 nM) and SMN proteins (EC1.5x: 87 nM).

Branaplam (LMI070; NVS-SM1) hydrochloride is a selective, orally active SMN2 splicing modulator that acts on SMN (EC50: 20 nM). branaplam hydrochloride inhibits hERG (IC50: 6.3 μM). In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride increased full-length SMN protein and improved survival.