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You are here:Home-Research Areas-Neurological Disease-Spinal Muscular Atrophy (SMA)

Request The Product List ofSpinal Muscular Atrophy (SMA) Spinal Muscular Atrophy (SMA)

Spinal Muscular Atrophy (SMA) is a group of genetic disorders in which a person cannot control the movement of their muscles due to a loss of nerve cells in the spinal cord and brain stem. It is a neurological condition and a type of motor neuron disease.

 Spinal muscular atrophy (SMA) causes muscle wasting and weakness. It can be difficult for a person with SMA to stand, walk, control their head movements, and even, in some cases, breathe and swallow. Some types of SMA are present from birth, but others appear later in life. Some types affect life expectancy.

Cat. No. Product Name CAS No. Information
H6869

Risdiplam

 		1825352-65-5

Risdiplam (RG7916, RG-7916, RO7034067) is a highly potent, selective, orally active?SMN2 splicing modifier for treatment of?spinal muscular atrophy (SMA); RG7916 is undergoing clinical trials across the spectrum of spinal muscular atrophy, where it has shown promising early results.
H6868

RG7800

 		1449598-06-4

RG7800 (RG-7800, RG 7800) is a potent, orally available, small molecule SMN2 splicing modifier with EC1.5X of 23 nM and 87 nM for SMN2 splicing and SMN2 protein, respectively; specifically modifies the alternative splicing of SMN2 exon 7 in SMA patient-derived cells and in two SMA mouse models; exhibits excellent pharmacokinetic and in vivo efficacy and has a favorable safety profile.
H6865

LMI-070

 		1562338-42-4

LMI070 (NVS-SM1, Branaplam ) is a potent SMN2 splice modulator (EC50=20 nM) that potential treatment of spinal muscular atrophy (SMA).
H6774

Verdiperstat

 		890655-80-8

Verdiperstat (AZD-3241) is a potent, selective, reversible, orally active myeloperoxidase (MPO) inhibitor with IC50 of 630 nM, >14-fold selectivity over thyroid peroxidase (TPO); inhibits PMA stimulate neutrophils and zymosan stimulated whole blood with IC50 of 80 nM, efficiently inhibits MPO activity in vivo during acute peritonitis in rats and has consistently shown neuroprotective efficacy in MPTP-lesioned mice, a model of Parkinson’s Disease; also demonstrates significant neuroprotection in MSA mouse model.
H6611

PF-DcpSi

 		2092917-19-4

A potent inhibitor of the mRNA decapping scavenger enzyme (DcpS) with IC50 of 0.11 nM; shows no significant activity for DHFR (IC50>30 uM); has in vivo mouse CNS PK profile, shows activity in SMA mouse model by prolonging survival and improving function thereby ruling out lysosomotropism.
H6245

CK-2127107

 		1345410-31-2

CK-2127107(CK-107, Reldesemtiv)?is a novel orally active fast skeletal troponin activator, selectively activates fast skeletal myofibrils with EC50 of 3.4 uM; has no effect on slow skeletal or cardiac myofibrils; significantly improves rotarod performance in exercise-intolerant LAD-HF rats.
H6094

Olesoxime

 		22033-87-0

Olesoxime (TRO 19622) is a cholesterol-like compound that binds directly to two components of the mitochondrial permeability transition pore: the voltage-dependent anion channel and TSPO (or PBR); rescues motor neurons from axotomy-induced cell death in neonatal rats and promoted nerve regeneration following sciatic nerve crush in vivio, significantly reduces established mechano-allodynia and mechano-hyperalgesia.
H6860

D-156844

 		1005501-84-7

D156844 is a potent SMN2 inducer and DcpS inhibitor; highly potent at activating SMN2 promoter activity with EC50 of 4 nM; increases SMN expression in neonatal mouse neural tissues, delays motor neuron loss at PND11 and ameliorates the motor phenotype of SMNDelta7 SMA mice; orally active.

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