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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.

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




PF-DcpSi is 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.




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.




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.

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