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Cat. No. Product Name CAS No. Information
PC-61397

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.

Spinal Muscular Atrophy (SMA)
 
Phase 2 Discontinued
PC-60222

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.

Spinal Muscular Atrophy (SMA)

Phase 2 Clinical

PC-45659

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).

Spinal Muscular Atrophy (SMA)

Phase 2 Clinical

PC-42423

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.

Spinal Muscular Atrophy (SMA)

Phase 2 Clinical

PC-63145

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.

Spinal Muscular Atrophy (SMA)

Phase 2 Discontinued

PC-62134

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.

Spinal Muscular Atrophy (SMA)

Phase 3 Clinical

PC-60223

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.

Spinal Muscular Atrophy (SMA)

Preclinical

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