Abstract: Described herein are neuroactive steroids of the Formula (I): or a pharmaceutically acceptable salt thereof, wherein , A, R1, R2, R3a, R4a, R4b, R5, R7a, and R7b are as defined herein. Such compounds are envisioned, in certain embodiments, to behave as GABA modulators. The present invention also provides pharmaceutical compositions comprising a compound of the present invention and methods of use and treatment, e.g., such for inducing sedation and/or anesthesia.

Abstract: Methods for designing heterobifunctional small molecules which selectively degrade/disrupt CDK4/6 and compositions and methods of using such degraders/disruptors to treat CDK4/6-mediated cancer are provided.

Abstract: The present invention provides compounds useful for the treatment of narcolepsy or cataplexy in a subject in need thereof. Related pharmaceutical compositions and methods are also provided herein.

Abstract: The present invention is related to a novel pyrimidine derivative compound or a pharmaceutically acceptable salt thereof, a process for preparing the same, and a pharmaceutical composition using the same.

Abstract: Among others, the present invention provides novel methods for extending lifespan or improving healthspan in a mammal, which include administration to the mammal of a composition containing a therapeutically effective amount of ergothioneine, or a pharmaceutically acceptable salt, acid, ester, analog or derivative thereof.

Abstract: This application discloses a new class of fused tricyclic heterocycles of formula (I), preparation methods thereof, pharmaceutical compositions comprising these compounds, and pharmaceutically acceptable salts, solvates, or prodrugs thereof, and their uses for the treatment of diseases in which modulation of STING is beneficial, for example, cancers, pre-cancerous disorders, and viral infections.

Abstract: The present invention relates to pharmaceutical agents useful for therapy and/or prophylaxis in a mammal, and in particular to azepane compounds, pharmaceutical composition comprising such compounds, and their use as menin/MLL protein/protein interaction inhibitors, useful for treating diseases such as cancer, myelodysplastic syndrome (MDS) and diabetes.

Abstract: Described herein are neuroactive steroids of the Formula (I): or a pharmaceutically acceptable salt thereof; wherein , A, R1, R2, R3a, R4a, R4b, R5, R7a, and R7b are as defined herein. Such compounds are envisioned, in certain embodiments, to behave as GABA modulators. The present invention also provides pharmaceutical compositions comprising a compound of the present invention and methods of use and treatment, e.g., such for inducing sedation and/or anesthesia.

Abstract: Disclosed are methods of treating a disorder or disease associated with myotonic dystrophy. Methods of treating a CNS dysfunction and/or cognitive impairment associated with myotonic dystrophy in a subject comprising administering a therapeutically effective amount of a GABAA receptor antagonist or inverse agonist to the subject are disclosed. Methods of treating a myotonic dystrophy associated disease or disorder caused by mis-splicing of GABRG2 in a subject comprising administering a therapeutically effective amount of a GABAA receptor antagonist or inverse agonist to the subject are disclosed. Methods of improving cognitive function or alertness in a subject having myotonic dystrophy comprising administering a therapeutically effective amount of a GABAA receptor antagonist or inverse agonist to the subject are disclosed.

Abstract: Methods for designing bivalent compounds which selectively degrade/disrupt EZH2 and compositions and methods of using such degraders/disruptors to treat EZH2-mediated cancer are provided.

Abstract: The present disclosure relates to crystalline forms of the compound 4-[(7-methoxy-2,3-dihydro-1,4-benzothiazepin-4(5H)yl)methyl]benzoic acid hemifumarate, pharmaceutical compositions comprising these compounds and uses thereof to treat diseases and conditions associated with Ryanodine Receptor (RyR) dysfunction, in particular cardiac and musculoskeletal disorders and diseases.

Abstract: Glutathione deficiencies and deficiencies in glutathione synthetase activity, and therapeutic methods for the treatment thereof.

Abstract: Methods of safely administrating a gut-selective JAK3 inhibitor by oral administration are described. Also described are methods for providing clinically proven safe treatment of gastrointestinal inflammatory diseases, such as celiac disease, by oral administration of a gut-selective JAK3 inhibitor.

Abstract: Disclosed herein are kinase inhibitory compounds, such as a receptor-interacting protein-1 (RIP1) kinase inhibitor compounds, as well as pharmaceutical compositions and combinations comprising such inhibitory compounds. The disclosed compounds, pharmaceutical compositions, and/or combinations may be used to treat or prevent a kinase-associated disease or condition, particularly a RIP1-associated disease or condition.

Abstract: A compound of formula (II), a tautomer thereof or a pharmaceutically acceptable salt thereof, and use thereof in the preparation of medicaments for treating solid tumor-related diseases.

Abstract: A method for purifying crude rocuronium bromide includes removing the residual solvent in crude rocuronium bromide by vacuum microwave drying or fluidized drying to meet medicinal requirements. High-quality rocuronium bromide can be obtained by the method of the present application. The method makes the large-scale industrial production of rocuronium bromide easy to realize, and can also avoid or reduce the use of diethyl ether which is a flammable and explosive solvent. In addition, the equipment used requires less expense and occupies less space.

Abstract: Disclosed are effective fixed dose oral testosterone undecanoate compositions for use in regimens without dose titration and methods for treatment of patients in need of testosterone replacement therapy. Also disclosed are criteria for continuation and discontinuation of such regimens, based on single serum T concentration levels measured at a steady state at predetermined times after initiation of said regimen and at a predetermined number of hours after administration of a morning dose of the regimen.

Abstract: The invention provides compositions and methods for treatment of proliferative vitreoretinopathy.

Abstract: A refining process includes steps of subjecting crude caprolactam to a first evaporative crystallization and a first solid-liquid separation to obtain a first caprolactam crystal and a first crystallization mother liquor; washing the first caprolactam crystal to obtain a second caprolactam crystal; optionally concentrating the first crystallization mother liquor to perform a second evaporative crystallization and a second solid-liquid separation to obtain a third caprolactam crystal and a second crystallization mother liquor; subjecting the third caprolactam crystal to a second washing to obtain a fourth caprolactam; optionally concentrating the second crystallization mother liquor to perform thermostatic crystallization, performing separation to obtain a fifth caprolactam crystal and a third crystallization mother liquor; washing the fifth caprolactam crystal to obtain a sixth caprolactam crystal; and subjecting the second caprolactam crystal to a hydrogenation reaction.

Abstract: The present invention provides a compound represented by the following formula (I): wherein, ring A is a substituted or unsubstituted non-aromatic heterocycle; ring C is a benzene ring or the like; Q is a 5-membered aromatic heterocycle or the like; each R1 is independently halogen or the like; L is substituted or unsubstituted alkylene; R3 is substituted or unsubstituted alkyl or the like; R4 is hydrogen or the like; and n is an integer of 1 to 3.