Abstract: The present invention provides a novel prophylactic and/or therapeutic agent for amyotrophic lateral sclerosis. Provided is a prophylactic and/or therapeutic agent for amyotrophic lateral sclerosis.

Abstract: The specification relates to imidazo [4,5-c] cinnolin-2-one compounds and pharmaceutically acceptable salts thereof, which selectively modulate ataxia telangiectasia mutated (“ATM”) kinase. The specification also relates to pharmaceutical compositions comprising one or more of the compounds and salts thereof as an active ingredient, and to the use of the compounds and salts thereof in the treatment of ATM-associated diseases or conditions, including cancers.

Abstract: The present disclosure generally relates to pharmaceutical compositions comprising N-(4-((4-(2-(3-chloro-4-(2-chloroethoxy)-5-cyanophenyl) propan-2-yl)phenoxy)methyl)pyrimidin-2-yl)methanesulfonamideN-(4-((4-(2-(3-chloro-4-(2-chloroethoxy)-5-cyanophenyl) propan-2-yl)phenoxy)methyl)pyrimidin-2-yl)methanesulfonamide or a pharmaceutically acceptable salt, solvate, stereoisomer, or prodrug thereof. In particular, the present disclosure relates to solid dispersion pharmaceutical compositions useful for treatment of various cancers, for example breast cancer and prostate cancer.

Abstract: The present disclosure relates to the use of stimulators of soluble guanylate cyclase (sGC), pharmaceutically acceptable salts thereof and pharmaceutical formulations or dosage forms comprising them, alone or in combination with one or more additional agents, for the treatment of various CNS diseases, wherein an increase in sGC stimulation, or an increase in the concentration of nitric oxide (NO), or cyclic guanosine 3?,5?-monophosphate (cGMP) or both, or an upregulation of the NO-sGC-cGMP pathway is desirable. Compounds useful in the methods of the invention are those of Formula (I) or pharmaceutically acceptable salts thereof.

Abstract: The present disclosure relates to methods for treating Parkinson's disease in a subject with a compound provided herein, pharmaceutical compositions comprising the compound, as well methods for monitoring subject's response to the treatment.

Abstract: Disclosed herein are compounds for binding and modulating the activity of cereblon (CRBN), and methods of use. The present invention also provides compounds that can be used as synthetic intermediates in the preparation of bifunctional compounds for use in targeted protein degradation. The present compounds are thus useful for the treatment or prophylaxis of tumors and cancers.

Abstract: Provided herein is a method of treating, preventing, or ameliorating one or more symptoms of a malignant solid tumor with a pyrazolylpyrimidine, e.g., a compound of Formula (I), or an enantiomer, a mixture of enantiomers, a diastereomer, a mixture of two or more diastereomers, a tautomer, a mixture of two or more tautomers, or an isotopic variant thereof; or a pharmaceutically acceptable salt, solvate, hydrate, or prodrug thereof.

Abstract: The use of heterocyclic inhibitors of ERK1/2 for treating cancer, specifically solid tumors. In particular, the ERK1/2 N inhibitor is (S)—N-(2-amino-1-(3-chloro-5-fluorophenyl)ethyl)-1-(5-methyl-2-((tetrahydro2H-pyran-4-yl)amino)pyrimidin-4-yl)-1H-imidazole-4-carboxamide which is administered to a subject twice a day, once a week and is used to treat cancers including non-small cell lung cancer (NSCLC), melanoma, pancreatic cancer, or colorectal cancer (CRC). In some embodiments, the cancer is a MAP-Km/MAPKi-naive pan tumor.

Abstract: The present disclosure features methods and related compositions that, inter alia, modulate the mitochondrial lipid shunt pathway, including the modulating the cellular mechanisms by which lipids (e.g., fatty acids) are transported between the mitochondria, stress granules, and lipid droplets.

Abstract: The present invention relates to a pharmaceutical composition for preventing or treating cancer, containing 2,6-dichloro-4-(4-(4-hydroxycyclohexylamino)-7H-pyrrolo[2,3-D]pyrimidin-5-yl)phenol as an active ingredient. It has been ascertained that the present invention inhibits the formation of mammospheres in a breast cancer cell line and, when compared to anticancer drugs sorafenib and etoposide, which are topoisomerase inhibitors widely used in lung cancer, ovarian cancer, colon cancer, melanoma and the like, exhibits remarkable effects greater than or equal to those of the anticancer drugs sorafenib and etoposide. In addition, a compound of example 1, according to the present invention, exhibits synergistic anticancer effects when combined with radiotherapy or other anticancer drugs in a breast cancer cell line and a liver cancer cell line, and thus can be developed as an anticancer drug or a food exhibiting excellent effects in the treatment of cancer.

Abstract: A combinatorial targeted therapy method for treating cancer, including metastatic cancer in a subject is provided, the method being designed to prevent unacceptable level of systemic toxicity in the subject and thus forced stoppage of the treatment, by performing initial molecular diagnostics to detect genomic alterations at each cancer site of the subject; for each cancer site, designing an initial combination targeted therapy by selecting a plurality of targeted drugs, based on the results of the initial molecular diagnostic at each cancer site; assigning each targeted drug to systemic or local delivery method, based on each targeted drug's properties; simultaneously treating all cancer sites according to the designed initial combination targeted therapy for each site, by delivering each targeted drug according to assigned delivery method to each targeted drug; and monitoring the progress of the cancer at each cancer site by performing follow-up molecular diagnostics at each cancer site.

Abstract: Disclosed are methods for treating NMDA receptor-mediated disorders by administering certain NR2B subunit-selective NMDA (N methyl-D aspartate) antagonists. NMDA receptor-mediated disorders include autism spectrum disorders, obsessive-compulsive disorder and anxiety disorders.

Abstract: The disclosure relates to Larotrectinib and salts thereof for use in a method for treating a patient having a cancer characterized by a neurotrophic receptor kinase gene fusion, wherein the patient has received and will receive a moderate CYP3A4 inducer to improve the anti-cancer efficacy of larotrectinib and salts thereof in patients taking one or more CYP3A4 modulating drugs.

Abstract: Modified release formulations, such as solid oral dosage forms comprising a core composition comprising Compound (I) and/or a pharmaceutically acceptable salt thereof; a sub-coating layer coating the core composition, said sub-coating layer comprising a polyvinyl alcohol and/or a hydroxypropyl methyl cellulose; and an enteric coating layer encapsulating the sub-coating layer and the core composition, said enteric coating layer comprising at least one polymer selected from an acrylic/methacrylic/ethacrylic acid homopolymer and copolymers thereof, a cellulose derivative, and a polyvinylpyrrolidone, and methods of administration of a Bruton's tyrosine kinase (BTK) inhibitor using said formulations.

Abstract: The present invention relates to a combination comprising a neurotensin receptor binding compound and FOLFIRINOX for use for the treatment of a neurotensin receptor overexpressing tumour in a subject.

Abstract: Methods for treating and/or preventing drug-induced thrombocytopenia (DITP) and vaccine-induced thrombosis and thrombocytopenia syndrome (VITT) with certain BTK inhibitors and/or pharmaceutically acceptable salts thereof are provided.

Abstract: Liquid pharmaceutical compositions of tofacitinib or its pharmaceutically acceptable salts thereof are described, which are suitable for oral administration, and which are stable under varying storage conditions for extended periods of time. Methods of treating auto-immune disorders are also described, using the stable liquid pharmaceutical compositions of tofacitinib. A stable liquid pharmaceutical composition of tofacitinib according to the invention comprises of (a) tofacitinib at a concentration of about 1 mg/mL or more; (b) a pharmaceutically acceptable liquid vehicle; (c) at least one anti-oxidant; and (d) optionally one or more other pharmaceutically acceptable excipients.

Abstract: Provided herein is a pharmaceutical composition comprising two or more compounds, wherein each compound is independently a phosphodiesterase inhibitor, an adenosine receptor antagonist, a calcium channel blocker, a histamine H1-receptor agonist, a histamine H2-receptor agonist, a histamine H3-receptor antagonist, or a ?2-adrenoreceptor agonist. Also provided herein is a method of treating, preventing, or ameliorating a cardiovascular disease in a subject, comprising administering to the subject in need thereof two or more compounds, wherein each compound is independently a phosphodiesterase inhibitor, an adenosine receptor antagonist, a calcium channel blocker, a histamine H1-receptor agonist, a histamine H2-receptor agonist, a histamine H3-receptor antagonist, or a ?2-adrenoreceptor agonist.

Abstract: The present invention relates to a crystalline form of a lamotrigine hydrate, a method for preparing the same and a composition comprising the same, and in particular, to a lamotrigine hydrate form A, a method for preparing the lamotrigine hydrate form A and a composition comprising the lamotrigine hydrate form A.

Abstract: Provided herein are methods of using 2-(2.6-dioxopiperidin-3-yl)-4-((2-fluoro-4-((3-morpholinoazetidin-1-yl)methyl)benzyl)amino)isoindoline-1, 3-dione, or an enantiomer, a mixture of enantiomers, a tautomer, an isotopolog, or a pharmaceutically acceptable salt thereof, in combination with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone or an equivalent thereof for treating, preventing or managing B-cell lymphoma.

Abstract: This disclosure provides a method of stabilizing the interaction of a Retinoic Acid Receptor-alpha (RAR?) and a corepressor, Nuclear Receptor Corepressor 1 (NCoR1) by contacting the RARa with an amount of a Chaperone Mediated Autophagy (CMA) Activator sufficient to stabilize the RAR?-NCoR1 interaction. Stabilizing the RAR?/corepressor interaction can prevent a neurodegenerative disorder in a subject at risk for developing the neurodegenerative disorder or slow the advancement of a neurodegenerative disorder in a subject having an early symptom or biomarker of the neurodegenerative disorder. The disclosure also provides a method of maintaining preventing or slowing the advancement of a retinal degenerative disorder in a subject having an early symptom or biomarker of the retinal degenerative disorder.

Abstract: The disclosure herein describes solubilizing an insoluble compound in water forming a complex with a hydrotrope and controlling the pH of the water or water-based solution. In some embodiments, methods for solubilizing a compound, such as meloxicam or piroxicam, in water are described. These methods can include forming a colloidal suspension of the compound, and then forming an aqueous colloidal suspension from the colloidal suspension.

Abstract: Provided are immediate or prolonged administration of certain potassium ATP (KATP) channel openers, optionally in combination with growth hormone, to a subject to achieve novel pharmacodynamic, pharmacokinetic, therapeutic, physiological, metabolic and compositional outcomes in the treatment of diseases or conditions involving KATP channels. Also provided are pharmaceutical formulations, methods of administration and dosing of KATP channel openers that achieve these outcomes and reduce the incidence of adverse effects in treated individuals. Further provided are methods of co-administering KATP channel openers with other drugs (e.g., in combination with growth hormone) to treat diseases of humans and animals (e.g., Prader-Willi Syndrome (PWS), Smith-Magenis syndrome (SMS), and the like.

Abstract: Provided herein are pharmaceutical compositions comprising midazolam and ketamine, and methods of inducing sedation (e.g., procedural sedation) in a subject using administration of such compositions, the compositions optionally including a pharmaceutically active compound of a third class. Compositions may be in sublingual or buccal form, or incorporated into vehicles for extended release. Methods for fabricating the compositions and using them for anesthesiological applications are also described.

Abstract: The invention relates to an apical sodium-dependent bile acid transport (ASBT) inhibitor for use in the treatment of renal diseases and disorders, such as cholemic nephropathy. Such treatment can include reducing serum bile acid concentrations, increasing urinary bile acids and improving liver as well as renal parameters.

Abstract: Biocompatible microparticles include an ophthalmically active cyclic lipid component and a biodegradable polymer that is effective, when placed into the subconjunctival space, in facilitating release of the cyclic lipid component into the anterior and posterior segments of an eye for an extended period of time. The cyclic lipid component can be associated with a biodegradable polymer matrix, such as a matrix of a two biodegradable polymers. Or, the cyclic lipid component can be encapsulated by the polymeric component. The present microparticles include oil-in-water emulsified microparticles. The subconjunctivally administered microparticles can be used to treat or to reduce at least one symptom of an ocular condition, such as glaucoma or age related macular degeneration.

Abstract: Disclosed herein are methods for topically treating strictures by orally administering corticosteroids. Dosages, formulations, and methods for administration of corticosteroids are provided.

Abstract: Described herein are methods of treating symptoms of neurodegenerative disorders using palmidrol and a cannabinoid.

Abstract: A pharmaceutical composition comprising: a compound of formula (1) and a polymer material, wherein the compound of formula (1) maintains a supersaturated concentration in the pharmaceutical composition and is suitable for storage at room temperature.

Abstract: The present disclosure relates to methods for treating poxvirus infections using a compound of Formula I: or a pharmaceutically acceptable salt or deuterated analog thereof.

Abstract: Compositions and methods that comprise 1) compounds with one or more substituted or unsubstituted boronic acid moieties (boronic acid compounds) and 2) one or more sulfur-containing compounds. Preferred compositions can exhibit reduced degradation over prolonged time storage periods.

Abstract: Provided are: a pharmaceutical composition, food composition, or feed composition for improving the intestinal microbial population comprising galactose, and for reducing body weight; and use thereof for preventing and/or treating and/or ameliorating obesity.

Abstract: Disclosed are methods for treating a cancer and/or enhancing immune responses to infiltration of tumors comprising administering to a subject a fucose. Also disclosed herein are methods of detecting the presence of a sugar-modified protein (i.e., a glycosylated protein).

Abstract: Provided are methods of treating a patient diagnosed with Fabry disease and methods of enhancing ?-galactosidase A in a patient diagnosed with or suspected of having Fabry disease. Certain methods comprise administering to a patient a therapeutically effective dose of a pharmacological chaperone for ?-galactosidase A, wherein the patient has a mutation in the nucleic acid sequence encoding ?-galactosidase A. Also described are uses of pharmacological chaperones for the treatment of Fabry disease and compositions for use in the treatment of Fabry disease.

Abstract: Disclosed is a viral receptor that contains a sialic acid compound at one side thereof to provide binding affinity to a virus, and contains a lipid at the other side thereof, and that can be widely used for the treatment of viral infections based on this characteristic.

Abstract: The subject invention relates to the prevention, treatment, or alleviation of symptoms, of infections caused by lipid enveloped viruses, preferably SARS-CoV-2 virus, preferably of COVID-19 disease, by the use of polyene macrolides, preferably nystatin. Treatment is preferably carried out by oral administration, but buccal or nasal spray can also be used. It may also be advantageous to administer the active ingredient rectally, preferably in the form of a rectal suppository.

Abstract: The present invention relates to a nutritional supplement for use to ameliorate the effect of a pneumonia in a human, which supplement comprises a mixture of bioflavonoids which contains at least 45% wt/wt (of the total bioflavonoids) of naringin and at least 15% wt/wt of neohesperidin, wherein said supplement is in a form suitable for oral administration or administration to the lung by insufflation or inhalation. The present invention also provides a method of treatment of pneumonia using the aforementioned nutritional supplements.

Abstract: The present disclosure provides methods of treating or preventing a SARS-CoV-2 infection or a SARS-CoV-2-infection associated disease or disorder (e.g. COVID-19) comprising administering to a subject in need thereof an effective amount of a pyrrolopyrimidine nucleoside analog of the Formula I, Formula IA, Formula IB or Formula II and pharmaceutical compositions thereof wherein Rc and A are defined herein.

Abstract: The use of inhibitors of tyrosine and threonine-specific cdc2-inhibitory kinase (Myt1) in the treatment of cancer is disclosed. In preferred embodiments, the Myt1 inhibitor is a carboxamide pyrrolopyrazine or carboxamide pyrrolopyridine of Formula I. The Myt1 inhibitors may be used in combination with a variety of other anti-cancer agents. Such agents include a WEE1 inhibitor, TOPI or TOP2A inhibitor, RRM1 or RRM2 inhibitor, AURKA or AURKB inhibitor, ATR inhibitor, TTK inhibitor, SOD1 or SOD2 inhibitor, BUB1 inhibitor, CDC7 inhibitor, SAE1 inhibitor, PLK1 inhibitor, UBA2 inhibitor, DUT inhibitor, HDAC3 inhibitor, CHEK1 inhibitor, MEN1 inhibitor, DOT1L inhibitor, CREBBP inhibitor, EZH2 inhibitor, PLK4 inhibitor, HASPIN inhibitor, METTL3 inhibitor, nucleoside analogs, and platinum-based DNA alkylating agents.

Abstract: An object of the present invention is to provide a pharmaceutical composition and an anti-tumor agent that exhibit an effect on a tumor having a decreased function of at least one of BAP1 or PBRM1. According to the present invention, there is provided a pharmaceutical composition for the treatment of a tumor having a decreased function of at least one of BAP1 or PBRM1, the pharmaceutical composition including 1-(2-deoxy-2-fluoro-4-thio-?-D-arabinofuranosyl)cytosine or a salt or prodrug thereof.

Abstract: The present invention relates to antiviral compounds selected from cytokinins, their nucleosides and nucleotide analogs, and their prodrugs as inhibitors of viral RNA synthesis, or their salts, solvates, derivatives, or even combinations of aforementioned compounds, for prophylactic treatment, curative (therapeutic) or mitigative coronavirus infection, represented by human and veterinary coronavirus, SARS-COV-2 and MHIV, and for the treatment of individuals potentially exposed to COVID-19. The present invention also comprises the methods for the manufacturing of such compounds, the antiviral pharmaceutical composition containing the compounds of the invention, as well as the use of the compounds, combinations of compounds, and method for the prophylactic, curative (therapeutic) or mitigative treatment of coronavirus infection, represented by coronavirus, in especial SARS-COV-2 and of patients with COVID-19, individual infected with SARS-COV-2 or potentially exposed to this virus.

Abstract: The present invention relates to a composition for expressing botulinum toxin in target tissue by injecting or topically applying an mRNA product capable of expressing the toxin.

Abstract: The invention relates to a combination therapy comprising an saRNA targeting C/EBP? and at least one additional active agent. Methods of using the combination therapy are also provided.

Abstract: Chondroitin sulfate-based pharmaceutical formulations useful for the treatment of ocular pain or discomfort, such as occurring from ocular surgery, dry eye disease, ocular surgery induced dry eye disease, contact lens induced ocular surface dry eye disease, preservative induced ocular surface disease, conjunctivitis, corneal abrasion, burns, blepharitis, and stye.

Abstract: The present invention provides a composition that controls pharmacokinetics. Specifically, the present invention provides: a composition for controlling pharmacokinetics, the composition containing a polyvalent cation as an active ingredient; and a method for controlling pharmacokinetics using the polyvalent cation.

Abstract: A colloidal suspension of single crystal cerium oxide nanoparticles for use in the treatment of a disorder or disease of the posterior segment of the eye by administering a topical ophthalmic dose of the colloidal suspension.

Abstract: A composition includes fibronectin peptides derived from the integrin-binding domain of fibronectin. The composition may inhibit self-activation of primary dendritic cells derived from lymphoid tissue isolated from the spleen. In a culture method, the activation of the primary dendritic cells cultured by adding the peptides can be normally induced by an immunostimulant. The primary lymphoid tissue dendritic cells activated while being cultured with the peptide combination enhance the proliferation of T cells and induce the production of cytokines such as interferon upon co-culture with T cells. The compound can be effectively used as a composition for the prevention or treatment of cancer, autoimmune diseases and inflammatory diseases, through dendritic cell culture, the induction of dendritic cell activation, and T cell proliferation by co-culture with dendritic cells.

Abstract: The current invention provides monocytic cells transfected with chimeric antigen receptor (CAR) to selectively home to tumors and upon homing differentiate into dendritic cells capable of activating immunity which is inhibitory to said tumor. In 5 one embodiment of the invention, monocytic cells are transfected with a construct encoding an antigen binding domain, a transcellular or structural domain, and an intracellular signaling domain. In one specific aspect of the invention, the antigen binding domain interacts with sufficient affinity to a tumor antigen, capable of triggering said intracellular domain to induce an activation signal to induce monocyte differentiation into DC.

Abstract: Provided herein are methods, compositions, and uses for treating subjects with diseases and conditions, such as those involving or associated with B cell maturation antigen (BCMA). In some aspects, the present disclosure relates to certain combination therapies that include administration of a cell therapy, such as a BCMA-targeted T cell therapy, in combination with immunomodulatory agents and other agents, such as dexamethasone, including for the treatment of subjects with multiple myeloma. In some embodiments, the multiple myeloma is a relapsed or refractory multiple myeloma.

Abstract: The present disclosure generally relates to a method of treating cancer by administration of autologous tumor infiltrating lymphocytes (TILs) isolated from a subject that has received prior treatment with an anti-cancer therapy that comprises an anti-clusterin antibody or antigen binding fragment thereof. The method of the present disclosure comprises administering the anti-cancer therapy to the subject, isolating TILs. and reinfusing TILs to the subject. The present disclosure also relates to the use of an anti-clusterin antibody or antigen binding fragment thereof in an in vitro or ex vivo method of generating tumor infiltrating lymphocytes (TILs).