Abstract: The present disclosure relates to compounds and methods for modulating the expression of fxn, and treating diseases and conditions in which fxn plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a trinucleotide repeat sequence GAA; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence GAA; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.

Abstract: A compound of formula (I) or an optical isomer thereof, and pharmaceutically acceptable salts, prodrugs, aquo-complexes or non-aqueous-solvent complexes thereof are provided. Experiments prove that, compared with a control compound MGL-3196, the compound of formula (I), which is obtained through specific substitution sites and specific substitution types, is higher in agonist activity to THR-beta and significantly improved in selectivity on THR-beta/THR-alpha. The compound can be used in preparing THR-beta agonist and drugs for treating adaption diseases (including dyslipidemia, hypercholesteremia, non-alcoholic steatohepatitis and non-alcoholic fatty liver disease) applicable to the THR-beta agonist.

Abstract: The present disclosure relates to transcription modulator molecules having a first terminus, a second terminus, and an oligomeric backbone and methods for treating Huntington's disease.

Abstract: The present disclosure relates to compounds and methods for modulating the expression of fxn, and treating diseases and conditions in which fxn plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence CGG; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence CGG; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.

Abstract: The present disclosure relates to transcription modulator molecules having a first terminus, a second terminus, and an oligomeric backbone and methods for treating Huntington's disease.

Abstract: The disclosure is directed to deutetrabenazine analogs, compositions comprising same and methods of detecting same in compositions comprising deutetrabenazine.

Abstract: The present invention relates to new extended release pharmaceutical compositions and methods of use thereof for the treatment of disorders.

Abstract: The present disclosure relates to compounds and methods which may be useful for modulating the expression of a target gene comprising a CGG trinucleotide repeat sequence and treating diseases and conditions in which the target gene plays an active role. The present disclosure provides compounds and methods for modulating the expression of fmr1 and fmr2, and provides compounds and methods for treating fragile X syndrome and fragile XE syndrome.

Abstract: The present invention relates to new pharmaceutical compositions comprising benzoquinoline compounds, and methods to inhibit vesicular monoamine transporter 2 (VMAT2) activity in a subject for the treatment of chronic hyperkinetic movement disorders.

Abstract: The present disclosure relates to compounds and methods for modulating the expression of dmpk, and treating diseases and conditions in which dmpk plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence CAG or CTG; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence CAG or CTG; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.

Abstract: A method for predicting a node state, including: obtaining static graphs and dynamic graphs of a plurality of nodes in a target network, where the static graphs and the dynamic graphs are all topology views; generating spatial feature data of the plurality of nodes based on the static graphs and the dynamic graphs; obtaining time feature data of the plurality of nodes; and obtaining a predicted state of a target node in a target time range based on the spatial feature data and the time feature data, where the target node is any node in the plurality of nodes. The method for predicting a node state provided in this application is applied to the field of node state prediction in a network, and uses a dynamic spatial feature in addition to a time feature and a static spatial feature. FIG.

Abstract: The present invention relates to new methods of manufacturing benzoquinoline inhibitors of vesicular monoamine transporter 2 (VMAT2), and intermediates thereof.

Abstract: A compound of formula (I) or an optical isomer thereof, and pharmaceutically acceptable salts, prodrugs, aquo-complexes or non-aqueous-solvent complexes thereof are provided. Experiments prove that, compared with a control compound MGL-3196, the compound of formula (I), which is obtained through specific substitution sites and specific substitution types, is higher in agonist activity to THR-beta and significantly improved in selectivity on THR-beta/THR-alpha. The compound can be used in preparing THR-beta agonist and drugs for treating adaption diseases (including dyslipidemia, hypercholesteremia, non-alcoholic steatohepatitis and non-alcoholic fatty liver disease) applicable to the THR-beta agonist.

Abstract: An aromatic amine androgen receptor (AR) and BET targeting protein degradation chimera compound is represented by formula I. Experimental results show that the compound can target and degrade both AR and BRD4, and down-regulate the expression of AR and BRD4 proteins; the compound can inhibit the proliferation of a variety of prostate cancer cells; the compound can inhibit the proliferation of a prostate cancer cell line LNCaP/AR, which overexpresses the AR, and can achieve a good inhibition effect on a prostate cancer cell line 22RV1, which is resistant to a marketed prostate cancer drug (enzalutamide). The compound also shows good metabolic stability, and has a good application prospect in the preparation of an AR and/or BET protein degradation targeting chimera, and a drug for the treatment of related diseases regulated by the AR and BET.

Abstract: A compound represented by formula (I), or a stereochemical isomer thereof, or a solvate thereof, or a pharmaceutically acceptable salt thereof, can inhibit the activity of histone acetylase p300 and inhibit the proliferation activity of a variety of tumor cells.

Abstract: The disclosure is directed to deutetrabenazine analogs, compositions comprising same and methods of detecting same in compositions comprising deutetrabenazine.

Abstract: The present invention relates to new dihydoxyphenyl modulators of neurotransmitter levels, pharmaceutical compositions thereof, and methods of use thereof.

Abstract: The disclosure is directed to deutetrabenazine analogs, compositions comprising same and methods of detecting same in compositions comprising deutetrabenazine.

Abstract: The disclosure is directed to deutetrabenazine analogs, compositions comprising same and methods of detecting same in compositions comprising deutetrabenazine.

Abstract: The present invention relates to new benzoquinolone inhibitors of VMAT2, pharmaceutical compositions thereof, and methods of use thereof.