Abstract: Described herein are antifungal peptoids, the development and characterization of the antifungal peptoids, methods of making the antifungal peptoids, and methods of using the antifungal peptoids. In some embodiments, the antifungal peptoids may be administered to a subject infected with or at risk of being infected with pathogenic fungi including, for example Cryptococcus spp. In some embodiments, the Cryptococcus spp. may include C. neoformans or C. gattii or both.

Abstract: The invention provides methods of treating or preventing an ocular disease or disorder in a subject, methods of treating or preventing ocular pain or discomfort comprising, administering to the subject a composition comprising a pharmaceutically acceptable carrier and a pharmaceutically effective amount of a transient receptor potential melastatin 8 (TRPM8) antagonist. In certain preferred embodiments, the ocular disease or disorder is a dry eye disease.

Abstract: The application discloses circRNAs as new biomarkers for the diagnosis of heart failure, the prediction of the clinical evolution of heart failure and/or prediction of the response to a treatment in a patient; methods for the prediction of outcome and diagnosis of heart failure and/or predicting the response to a treatment are provided based on measuring said one or more circRNAs; and kits and devices for measuring said circRNAs and/or performing said methods. Further provided are methods for treating patients with heart failure based on the evaluation of said one or more circRNAs.

Abstract: Provided herein are compositions and methods for administration of sulcardine to a subject in need thereof.

Abstract: The invention relates to methods of treating depression with Compound 1 or pharmaceutically acceptable salts thereof.

Abstract: Anti-viral compounds with low cytotoxicity are identified from screening of products found in Red Sea sponges, including the sponge Stylissa carteri. The identified compounds can be brominated pyrrole-2-aminoimidazole alkaloids and derivatives thereof. Specific examples of identified compounds include oroidin, hymenialdisine, and debromohymenialdisine, as well as derivatives thereof. The compounds also can be useful scaffolds or pharmacores for further chemical modification and derivatization. Selected compounds, particularly oroidin, show selective anti-viral HIV-1 activity coupled with reduced cytotoxicity. The compounds can function as HIV reverse-transcriptase inhibitors, and molecular modeling can be used to confirm inhibition.

Abstract: Aspects of the disclosure include methods for treating hyperhidrosis in a subject with a composition including a muscarinic antagonist and a muscarinic agonist. In practicing methods according to certain embodiments, a therapeutically effective amount of a composition including a muscarinic antagonist or a pharmaceutically acceptable salt thereof and a muscarinic agonist or a pharmaceutically acceptable salt thereof is administered to a subject and is sufficient to reduce hyperhidrosis in the subject and to reduce a dry mouth side effect of the muscarinic antagonist. Compositions for practicing the subject methods are also described as well as dose units containing one or more of the subject compositions.

Abstract: A method for treating acute myeloid leukemia (AML), such as poor risk AML, by administering to a subject in need thereof an effective amount of a mitochondrial activity inhibitor, for example a class A electron transport chain (ETC) complex I inhibitor such as Mubritinib or a pharmaceutically acceptable salt thereof, is disclosed. The AML to be treated may be characterized by certain features, such as high level of expression of one or more Homeobox (HOX)-network genes, high and/or low expression of specific genes, the presence of one or more cytogenetic or molecular risk factors such as intermediate cytogenetic risk, Normal Karyotype (NK), mutated NPM1, mutated CEBPA, mutated FLT3, mutated DNMT3A, mutated TET2, mutated IDII1, mutated IDII2, mutated RUNX1, mutated WT1, mutated SRSF2, intermediate cytogenetic risk with abnormal karyotype (intern(abnK)), trisomy 8 (+8) and/or abnormal chromosome (5/7), and/or a high leukemic stem cell (LSC) frequency.

Abstract: Provided herein are methods of treating, preventing, ameliorating or managing a nonsense mutation mediated epileptic disease, comprising administering a 1,2,4-oxadiazole benzoic acid to a patient having a nonsense mutation mediated epileptic disease. In particular, provided herein are methods of treating, preventing, ameliorating or managing a CDKL5 and/or SCN1A (Dravet syndrome) nonsense mutation mediated epileptic disease.

Abstract: The present disclosure provides pharmaceutical combinations of orally administered irinotecan and a P-gp inhibitor. The pharmaceutical combinations are suitable for the treatment of cancer in a subject and for reducing or preventing toxicity, hypersensitivity-type infusion reactions, and other negative outcomes resulting from or associated with intravenously administered irinotecan (e.g., Camptosar®) therapy in a subject suffering from cancer.

Abstract: The technical field of the invention is in pharmaceutical compounds and methods. In an aspect, the disclosure provides macrolide compounds suitable for use as antifungal agents, as well as methods for their use and compositions containing the same.

Abstract: Treatment of non 24 sleep wake disorder using gaboxadol or a pharmaceutically acceptable salt thereof is provided. Pharmaceutical compositions that may be used to improve one or more symptoms of non 24 sleep wake disorder are provided.

Abstract: Disclosed herein is a method for modulating Program Death Receptor Ligand 1 (PDL1) in a cancer cell, comprising contacting the cell with a composition comprising a histone deacetylase (HDAC) inhibitor. Also disclosed is a method for treating a tumor in a subject, comprising administering to the subject a thereapeutically effective amount of a composition comprising a histone deacetylase (HDAC) inhibitor and a composition comprising a thereapeutically effective amount of a Program Death Receptor Ligand 1 (PDL1) inhibitor, a Programmed Death 1 receptor (PD1) inhibitor, or a combination thereof.

Abstract: This invention provides a medicament for the treatment of cancer, which cause a reduction of cancer. This invention relates to use of a compound which has inhibitory activities against prostaglandin E2 receptor (EP4 receptor) and is represented by the following general formula (I), (II), (III), or (IV), or a pharmaceutically acceptable salt thereof, or a pharmaceutical composition comprising the compound or the salt for the manufacture of a medicament for the treatment of cancer. The invention relates to a method for treatment of cancer comprising administering the compound or a pharmaceutically acceptable salt thereof, or a pharmaceutical composition comprising the compound or the salt to humans or animals. The compound or a pharmaceutically acceptable salt thereof, or the pharmaceutical composition may be used in combination with one or more second active agents.

Abstract: The disclosure relates to chemotherapy treatments and in particular, formulations for antiemetic therapy. Parenteral formulations comprising Palonosetron, NK1 receptor antagonist and a corticosteroid is provided. Also provided is a process of preparing such formulations.

Abstract: The invention provides methods for the treatment of myopia. The compositions preferably comprise aceclidine. The compositions optionally contain a surfactant, a viscosity agent, a polyol and/or a selective ?-2 adrenergic receptor agonist.

Abstract: The present disclosure provides inhibitors of activin receptor-like kinase 5 (ALK5). Also disclosed are methods to modulate the activity of ALK5 and methods of treatment of disorders mediated by ALK5.

Abstract: The disclosure is directed to methods of treating dyskinesia in cerebral palsy in human patients using deutetrabenazine and its active metabolites.

Abstract: The present invention relates to pharmaceutical compositions and combinations comprising regorafenib or its hydrate, solvate, metabolite or pharmaceutically acceptable salt or a polymorph thereof and a PD-1/PD-L1(2) inhibitor for treating, preventing or managing diseases and conditions including hyperproliferative disorders such as cancer in humans and other mammals.

Abstract: Provided herein are compositions and methods for treating, inhibiting and/or reducing the severity of neuroblastoma, small cell lung cancer (SCLC), large cell neuroendocrine cancer (LCNEC), large-cell carcinoma (LCC), squamous cell carcinoma (SqCC), and/or adenocarcinoma (AC) in subjects in need thereof. The methods include providing an agent that inhibits expression or activity of ONECUT2 and administering a therapeutically effective amount of the agent so as to treat, inhibit and/or reduce the severity of neuroblastoma, small cell lung cancer (SCLC), large cell neuroendocrine cancer (LCNEC), large-cell carcinoma (LCC), squamous cell carcinoma (SqCC), and/or adenocarcinoma (AC) in the subject.

Abstract: Provided is an anesthetic composition for locally administrating an amide-type anesthetic into a subject in need thereof. The anesthetic composition has multilamellar vesicles with entrapped amide-type anesthetic prepared by hydrating a highly entrapped lipid structure comprising an amide-type anesthetic and a lipid mixture with an aqueous buffer solution at a pH higher than 5.5. Also provided is a method to prepare an anesthetic composition using a simpler and more feasible process for large-scale manufacture and for providing a high molar ratio of amide-type anesthetic to phospholipid content as compared to the prior art. This anesthetic composition has a prolonged duration of efficacy adapted to drug delivery.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Disclosed is a method for enhancing tumor response to chemotherapy, the method comprising administering a short-acting anti-angiogenic agent (AAA) capable of activating ASMase to a subject afflicted with a solid tumor, and thereby creating a time interval of increased susceptibility of said tumor to one or more chemotherapeutic agents, followed by administration of at least one chemotherapeutic agent within the interval. The interval can be defined in terms of a short-duration activation of ASMase signaling by the AAA. Disclosed are also methods for predicting the tumor response in a patient afflicted with a solid tumor to a chemotherapeutic agent, using as an indicator of the response ASMase level or activity (or ceramide level) in the patient following the administration of the chemotherapeutic agent to the patient, or dynamic IVIM based DW-MRI to measure perfusion alterations following administration of the chemotherapeutic agent.

Abstract: The present invention aims to find a novel pharmaceutical use of omidenepag, esters thereof, or salts thereof. The present inventors have made intensive studies to find a novel pharmaceutical use of omidenepag, an ester thereof, or a salt thereof, and have found as a result that omidenepag, an ester thereof, or a salt thereof significantly dilates retinal blood vessels, and significantly increases blood flow. Therefore, omidenepag, an ester thereof, or a salt thereof is expected to protect retinal nerve cells and be useful for prevention and/or treatment of ophthalmic diseases involving retinal nerve cell disorder.

Abstract: An oral methylphenidate extended release tablet is described, which can be scored and still retain its extended release profile. The tablet contains a combination of an uncoated methylphenidate-ion exchange resin complex, a barrier coated methylphenidate-ion exchange resin complex-matrix, and an uncomplexed methylphenidate active component. Following administration of a single dose of the extended release methylphenidate chewable tablet, a therapeutically effective amount of methylphenidate is reached in less than about 20 minutes and the composition provides a twelve-hour extended release profile.

Abstract: The invention relates to medicine and concerns the treatment of diseases related to the aberrant activity of fractalkine and monocyte chemoattractant proteins 1-4 (CCL2, CCL7, CCL8, CCL13), preferably the treatment of pain, fever, pneumonia, bronchitis, bronchiolitis, alveolitis, rheumatoid arthritis, psoriasis and other diseases, using the compound 1-(2-(1H-imidazol-4-yl)ethyl)piperidine-2,6-dione or a pharmaceutically acceptable salt or solvate thereof The present invention also relates to pharmaceutical compositions that contain a therapeutically effective amount of the claimed compound according to the invention. The present compound and pharmaceutically acceptable salts thereof are highly effective in inhibiting the activity of the glutaminyl cyclase enzyme, which is involved in particular in processes of post-translational modification of the above-mentioned cytokines.

Abstract: The present invention relates to a pharmaceutical composition comprising a therapeutically effective dose of Lenvatinib mesylate having sodium wherein the weight ratio of Lenvatinib mesylate to sodium carbonates ranges from 1:1.5 to 1:5.

Abstract: The present invention provides methods of use of hexokinase 2 (HK2)/mitochondria-detaching compounds, including jasmonate derivatives and piperazine derivatives and pharmaceutical compositions including such compounds for inducing immune responses in a subject, including potentiating the immune response to hyperproliferative disorders such as cancer and potentiating the immune response to infectious diseases.

Abstract: A method for inducing programmed cell death in cancer cells and a pharmaceutical composition therefor are provided. Provided is a method for inducing programmed cell death in cancer cells, the method including enhancing production of nitric oxide in cancer cells. Provided is a pharmaceutical composition for inducing programmed cell death in cancer cells, the pharmaceutical composition containing, as an active component, a compound that enhances production of nitric oxide in cancer cells. Provided is use of a compound that enhances production of nitric oxide in cancer cells, in a method for preventing, improving, suppressing progression of, and/or treating cancers.

Abstract: Disclosed herein are methods for treating solid tumors by direct injection into the tumors of chemotherapeutic particles, methods for inhibiting tumor metastasis by administering chemotherapeutic particles to a subject having a tumor, and compositions that include chemotherapeutic particles, small amounts of a polysorbate, and a carrier.

Abstract: Disclosed herein are methods for treating solid tumors by direct injection into the tumors of chemotherapeutic particles, methods for inhibiting tumor metastasis by administering chemotherapeutic particles to a subject having a tumor, and compositions that include chemotherapeutic particles, small amounts of a polysorbate, and a carrier.

Abstract: The present invention includes methods for treating a proliferative disorder by blocking both PDGFR and VEGFR signaling comprising a therapeutically effective amount of crenolanib or salt in combination with a VEGF/VEGFR inhibitor that is not axitinib wherein the crenolanib, VEGF/VEGFR inhibitor that is not axitinib are provided at least one of sequentially or concomitantly, in a subject for use in the treatment of the proliferative disorder, wherein the subject is a human subject.

Abstract: The present disclosure provides pharmaceutical combinations of orally administered docetaxel and a P-gp inhibitor. The pharmaceutical combinations are suitable for the treatment of cancer in a subject and for reducing or preventing toxicity, hypersensitivity-type infusion reactions, and other negative outcomes resulting from or associated with intravenously administered docetaxel (e.g., Taxotere® or docetaxel formulated with polysorbate 80) therapy in a subject suffering from cancer.

Abstract: The invention generally relates to solid forms of berberine ursodeoxycholate, and to pharmaceutical compositions, and methods of preparation and therapeutic use thereof. In particular, the invention relates to solid forms of berberine ursodeoxycholate and pharmaceutical compositions thereof useful in treating and/or preventing various diseases or disorders, including metabolic disorders, heart diseases, neurodegenerative diseases and liver diseases.

Abstract: The present invention provides safe, low-dose, therapeutic combinations of CNS drugs with peripheral adrenergic receptor agonists, and their use in methods for treating various conditions.

Abstract: Methods and compositions are provided for preventing or reversing loss of the therapeutic effect of a drug, where the loss is associated with the repeated administration of the drug to a patient. The method includes administering to the patient a dopamine receptor agonist or partial agonist or a drug that increases the extracellular level of dopamine by enhancing release of dopamine, decreasing the removal of dopamine from the extracellular space, enhancing the synthesis of dopamine within the brain, or decreasing metabolic degradation of dopamine; and also administering to the patient an opioid receptor antagonist in an ultra-low dose amount, wherein the ultra-low dose amount is effective to prevent or reverse loss of therapeutic effects associated with the repeated administration of the drug to the patient. The methods are useful for various treatments, including treating Parkinson's Disease, Restless Leg Syndrome, depression, schizophrenia, psychostimulant drug abuse, or attention deficit disorder.

Abstract: The present invention concerns a pharmaceutical composition, advantageously a liquid suspension, comprising: a. temozolomide or a salt thereof; b. at least one agent controlling the solid state of temozolomide in suspension; c. a pharmaceutically acceptable liquid vehicle, advantageously water; d. optionally at least one acid in a quantity so that the pH of the composition is below 5; or a powder blend for reconstituting said suspension.

Abstract: The invention relates to vortioxetine hydrobromide having particle size distribution of D98 100-200?, D50 35-90? and D5 7-30? as well as to a stable, reproducible and bioequivalent pharmaceutical composition comprising vortioxetine hydrobromide having particle size distribution of D98 100-200?, D50 35-90? and D5 7-30? and one or more pharmaceutically acceptable excipient. It is produced by wet granulation techniques.

Abstract: The present invention discloses halimide and plinabulin and structural analogues and their use in the treatment and prevention in epilepsy and other seizures. The present invention further discloses methods to screen halimide-like molecules as pharmaceutically active compounds.

Abstract: Provided herein are pharmaceutical formulations comprising a monoacylglycerol lipase (MAGL) inhibitor, or a pharmaceutically acceptable salt thereof, and at least one pharmaceutically acceptable excipient.

Abstract: Aspects and embodiments of the present invention relate to the treatment of fatty liver diseases such as for example, Non-Alcoholic Fatty Liver Disease (NAFLD) and Non-Alcoholic Steatohepatitis (NASH). Particularly, but not exclusively certain embodiments relate to activators of glucokinase (GKAs) for use in the treatment of these fatty liver diseases. Also included in the present invention are inter alia pharmaceutical compositions comprising the GKAs, together with methods of treating such disorders as well as other subject matter.

Abstract: A composition and method for preventing or reducing the incidence of a transient ischemic attack in a subject at risk for developing a stroke comprises orally administering to the subject a prophylactically effective amount of a pharmaceutical composition comprising an ASIC1a inhibitor capable of penetrating the blood-brain barrier. Preferred ASIC1a inhibitors for use in the disclosed methods include amiloride and amiloride analogs.

Abstract: Certain embodiments are directed to methods of treating or preventing an addictive behavior in a subject by administering to the subject an effective amount of a 5-HT2C receptor agonist in combination with a DA D3/D4 receptor antagonist.

Abstract: The present invention provides for compounds of Formula I-I and embodiments and salts thereof for the treatment of diseases (e.g., neurodegenerative diseases). R1, R2, R3, X1, X2, A and Cy variable in Formula I-I all have the meaning as defined herein.

Abstract: There is a method of treating or preventing sarcoidosis-associated pulmonary hypertension in a patient. The method has the step of administering to the patient a therapeutically effective amount of one or more compounds: (S)-ethyl 8-(2-amino-6-((R)-1-(5-chloro-[1,1?-biphenyl]-2-yl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)-2,8-diazaspiro[4.5]decane-3-carboxylate or a pharmaceutically acceptable salt thereof, or (S)-8-(2-amino-6-((R)-1-(5-chloro-[1,1?-biphenyl]-2-yl)-2,2,2-trifluoroeth-oxy)pyrimidin-4-yl)-2,8-diazaspiro[4.5]decane-3-carboxylic acid or a pharmaceutically acceptable salt thereof, or a combination of the foregoing.

Abstract: There is a method of treating or preventing interstitial lung disease in a patient. The method includes the step of administering to the patient a therapeutically effective amount of the compound of the following formula: or a pharmaceutically acceptable salt thereof.

Abstract: The present invention relates to kinase inhibitor C8-C16 aliphatic sulfate salts, compositions containing kinase inhibitor C8-C16 aliphatic sulfate salts and uses thereof.

Abstract: In one aspect, the present invention provides a method for treating or ameliorating the effects of a HER2 positive cancer in a subject. In some embodiments, the method comprises administering a combination therapy comprising an anti-HER2 antibody and tucatinib. In some embodiments, the method further comprises administering a chemotherapeutic agent (e.g., an antimetabolite) to the subject. Pharmaceutical compositions and kits are also provided herein.

Abstract: The present invention relates generally to methods for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to methods for the use of a small-molecule autophagy modulator for treatment of ribosomal disorders and ribosomopathy. The invention also relates to small molecule drug discovery and methods of screening compositions to determine their effectiveness for treatment of ribosomal disorders and ribosomopathies.

Abstract: A method of treating exposure to organophosphate agents and preventing morbidity due to such exposure through the administration of a pyrazolopyrimidine compound such as ocinaplon, zaleplon, indiplon, or divaplon.