spinocerebellar

Rimtuzalcap (CAD-1883) (CAD-1883), a pioneering selective positive allosteric modulator of small-conductance calcium-activated potassium channels (SK channels), is employed in the study of movement disorders such as spinocerebellar ataxia (SCA) and essential tremor (ET).

NS13001 (N-(4-Chlorophenyl)-2-(3,5-dimethyl-1H-pyrazol-1-yl)-9-methyl-9H-purin-6-amine) is a potent, selective, orally active allosteric positive modulator of SK channels (small conductance calcium-activated potassium channels). The EC50s are 1.8 and 0.14 μM for SK2 and SK3, respectively. NS13001 holds promise as a potential therapeutic agent for treatment of spinocerebellar ataxia type 2 (SCA2) and possibly other cerebellar ataxias.

Troriluzole is an orally active glutamate modulator with anticancer activity.Troriluzole enhances the expression of excitatory amino acid transporters located in glial cells and is used in the study of spinocerebellar ataxia, Alzheimer's disease, and generalized anxiety disorder (GAD).

ATXN1-MED15 PPI-IN-1 is an inhibitor of the ATXN1/MED15 interaction. It binds to residues 99 to 163 of ATXN1, disrupting the interaction between ATXN1 and MED15, as well as the dimerization of polyQ-expanded ATXN1. This compound is applicable for research related to Spinocerebellar Ataxia Type 1.

ARN 11391 enhances the function of the IP3-gated calcium channel ITPR1 (Inositol 1,4,5-trisphosphate (IP3) receptor type 1), boosting intracellular calcium ion signaling by directly targeting ITPR1. Furthermore, ARN 11391 amplifies calcium signaling in cells with mutant ITPR1 linked to SCA29, a variant of spinocerebellar ataxia.