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Cat. No. Product Name CAS No. Information



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




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



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




D-156844 (D156844) is a potent, orally bioavailable SMN2 promoter activator with EC50 of 4 nM and 2.3-fold induction of the SMN2 promoter, a potent inhibitor of the mRNA decapping enzyme DcpS; up-regulates expression of the mouse SMN gene in NSC-34 cells, induces SMN in a dose-dependent manner in type 1 SMA patient fibroblasts; possesses desirable pharmaceutical properties, including excellent brain exposure and long brain half-life.




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




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



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

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