Abstract: Disclosed are compounds of the general formula (I), wherein R1-R3 are as defined herein, for use as MALT1 inhibitors in the treatment of autoimmune and inflammatory diseases or disorders. Methods of synthesizing the compounds are also disclosed. Also disclosed are pharmaceutical compositions containing a compound of the invention and a method of treating a patient for an autoimmune or an inflammatory disease or disorder, for example, a cancer, by administering a compound of the invention.

Abstract: Disclosed are novel calcium release-activated calcium (CRAC) channel inhibitors, methods for preparing them, pharmaceutical compositions containing them, and methods of treatment using them. The present disclosure also relates to methods for treating non-small cell lung cancer (NSCLC) with CRAC inhibitors, and to methods for identifying therapeutics for treating and of diagnosing cancer.

Abstract: The current invention is in the field of molecular biology/pharmacology and provides methods of using compounds that modulate the effects of GPR30/GPER for treating obesity and diabetes (preferably agonists) as well as disease states and/or conditions that result from excessive formation of reactive oxygen species (preferably antagonists). These compounds may function as agonists and/or antagonists of the disclosed estrogen receptor and/or modulate the expression/upregulation of nox and nox-associated reactive oxygen species (ROS).

Abstract: The present application relates to novel substituted 5,6,7,8-tetrahydro[1,2,4]triazolo[4,3-a]pyridin-3(2H)-ones and 2,5,6,7-tetrahydro-3H-pyrrolo[2,1-c][1,2,4]triazol-3-ones, to processes for preparation thereof, to the use thereof, alone or in combinations, for treatment and/or prophylaxis of diseases, and to the use thereof for production of medicaments for treatment and/or prophylaxis of diseases, especially for treatment and/or prophylaxis of lung inflammation disorders.

Abstract: The present invention concern a 2-oxo-1,2-dihydropyridine-3-carboxamide compound of Formula (I) in the treatment of pathologies which require a dual inhibitor of PDK1/AurA enzymes such as for instance tumours, particularly glioblastoma.

Abstract: The present invention provides methods for preventing or treating a fibrotic disease in a subject. In some embodiments, the fibrotic disease is fatty liver disease, non-alcoholic fatty liver disease, or non-alcoholic steatohepatitis. In particular aspects, the methods comprise administering a small molecule inhibitor of Shc to achieve pharmacological suppression of Shc protein activity in the subject.

Abstract: The present invention relates to methods of treating patients with advanced forms of cancer, such as unresectable or metastatic renal cell carcinoma or kidney cancer, in which X4P-001 or a pharmaceutically acceptable salt thereof is administered as monotherapy or in combination with an immune checkpoint inhibitor, such as nivolumab. The methods demonstrate surprising results, including regression of disease, with comparatively little toxicity.

Abstract: The invention provides methods of reducing development of mild cognitive impairment and/or learning disorders and/or memory disorders in a subject suspected to have or suffering from Alzheimer's disease (AD). In some embodiments, the methods comprise administering a therapeutically effective amount of a beta-2 nicotinic acetylcholine receptor (n AChR) antagonist and/or beta-2 nAChRnegative allosteric modulator (NAM) to the subject over a therapeutic dosing period to thereby reduce the rate of development of mild cognitive impairment and/or learning disorders and/or memory disorders in the subject over the therapeutic dosing period.

Abstract: The present invention relates to injectable, extended-release, pharmaceutical formulations comprising a nalbuphine ester prodrug homogenously dissolved in a solution comprising a pharmaceutically acceptable oil and an oil-miscible retaining solvent, as well as manufacturing processes and medical uses of the formulations. The invention further provides methods for adjusting the duration of action of the formulations by varying the ratio of the pharmaceutically acceptable oil and the oil-miscible retaining solvent.

Abstract: The present invention relates to a pharmaceutical combination which may be useful for the treatment of diseases which involve cell proliferation, especially metastatic colorectal cancer comprising 3-Z-[1-(4-(N-((4-methyl-piperazin-1-yl)-methylcarbonyl)-N-methyl-amino)-anilino)-1-phenyl-methylene]-6-methoxycarbonyl-2-indo lino ne or a pharmaceutically acceptable salt thereof, and 2?-deoxy-5-(trifluoromethyl)uridine or a pharmaceutically acceptable salt thereof, and 5-chloro-6-[(2-iminopyrrolidin-1-yl)methyl]pyrimidine-2,4(1H,3H)-dione or a pharmaceutically acceptable salt thereof, wherein the molar ratio of 2?-deoxy-5-(trifluoromethyl)uridine or a pharmaceutically acceptable salt thereof, and 5-chloro-6-[(2-iminopyrrolidin-1-yl)methyl]pyrimidine-2,4(1H,3H)-dione or a pharmaceutically acceptable salt thereof, is 1:0.

Abstract: The present invention relates to a combination of pure 5-HT6 receptor antagonist, acetylcholinesterase inhibitor and NMDA receptor antagonist. Also, the present invention provides pure 5-HT6 receptor (5-HT6R) antagonists, or the pharmaceutically acceptable salt(s) thereof in combination with or as adjunct to acetylcholinesterase inhibitor and N-Methyl-D-aspartate (NMDA) receptor antagonist and their use in the treatment of cognitive disorders. The invention further provides the pharmaceutical composition containing the said combination.

Abstract: Disclosed herein are methods for treating a cancer and a method for sensitizing a cancer to a chemotherapeutic agent. Each method administers an agent that selectively inhibits HDAC1 and HDAC2 to a subject in need thereof. Also disclosed herein are methods for determining if a cancer is sensitive to an agent that selectively inhibits HDAC1 and HDAC2 and methods for monitoring the efficacy of a treatment for a cancer that includes administration of an agent that selectively inhibits HDAC1 and HDAC2.

Abstract: Compounds of formula (I) or their enantiomers, diastereoisomers thereof and the addition salts thereof with pharmaceutically acceptable bases or acids, for use for the prevention or treatment, preferably treatment, of NASH.

Abstract: Methods for treating a CB2 receptor-related disorder (e.g., pain, fibrosis) are provided. These methods are directed to reducing the risk of adverse events based on reduced blood pressure and/or heart rate in subjects in need of treatment with a CB2 receptor agonist compound (e.g., APD371).

Abstract: The application discloses methods and compositions for regulating neuronal activities in the brain that are useful, for example, for treating metabolic disorders.

Abstract: Provided are substituted benzaldehydes and derivatives thereof that act as allosteric modulators of hemoglobin, methods and intermediates for their preparation, pharmaceutical compositions comprising the modulators, and methods for their use in treating disorders mediate by hemoglobin and disorders that would benefit from increased tissue oxygenation.

Abstract: The present invention relates to novel 6,7-dihydropyrazolo[1,5-a]pyrazin-4(5H)-one derivatives as negative allosteric modulators (NAMs) of the metabotropic glutamate receptor subtype 2 (“mGluR2”). The invention is also directed to pharmaceutical compositions comprising such compounds, to processes for preparing such compounds and compositions, and to the use of such compounds and compositions for the prevention or treatment of disorders in which the mGluR2 subtype of metabotropic receptors is involved.

Abstract: The present invention provides a compound of formula IA or a pharmaceutically acceptable salt thereof; a method for manufacturing the compounds of the invention, and its therapeutic uses. The present invention further provides a combination of pharmacologically active agents and a pharmaceutical composition.

Abstract: The present invention relates to compounds of formula (I) and pharmaceutically acceptable salts thereof, pharmaceutical compositions comprising the compounds of formula (I) or their pharmaceutically acceptable salts, and methods of using said compounds, salts and compositions in the treatment of various disorders associated with CRM1 activity.

Abstract: A method of treating skeletal muscle cachexia and inflammation associated with burn injury in a subject in need thereof is provided, the method including administering to the subject an effective amount of a phosphodiesterase-4B (PED4B)-selective inhibitor.

Abstract: A hair growth formulation includes minoxidil and foeniculum vulgare, atriplex nummularia extract, or a combination of foeniculum vulgare and atriplex nummularia extract. A method for increasing hair growth includes applying a hair growth formulation to a subject.

Abstract: A drug containing, as an active ingredient, a compound represented by ALK inhibitors such as brigatinib, AP26113-analog, and AZD3463 has been found to be effective against a non-small cell lung cancer having a point mutation at C797S in EGFR which has acquired a resistance to chemotherapy agents. Further, the drug used in combination with an anti-EGFR antibody demonstrates a notable suppression effect on the tumor growth. The drug has a potential to be a therapeutic agent effective against a non-small cell lung cancer which is resistant to gefitinib, a first generation therapeutic agent and osimertinib, a third generation therapeutic agent.

Abstract: A novel therapeutic agent and novel anti-HTLV-1 drug for HTLV-1-associated myelopathy/tropical spastic paraparesis (HAM/TSP) is provided. A substance capable of inhibiting tyrosine kinase encoded by the ABL1 gene is contained as an active ingredient.

Abstract: Disclosed is a method of treating in a subject hair loss disorders that are beneficially treated by administering a JAK1 and/or JAK2 inhibitor. The method comprises administering to the subject an amount in the range of about 4 mg to about 50 mg of Compound (I), or a pharmaceutically acceptable salt thereof. This invention also provides compositions comprising Compound (I) and the use of such compositions in the described methods.

Abstract: The disclosure relates to a pharmaceutical combination of a mdm2/4 inhibitor, namely (S)-1-(4-Chloro-phenyl)-7-isopropoxy-6-methoxy-2-(4-{methyl-[4-(4-methyl-3-oxo-piperazin-1-yl)-trans-cyclohexylmethyl]-amino}-phenyl)-1,4-dihydro-2H-isoquinolin-3-one or (S)-5-(5-Chloro-1-methyl-2-oxo-1,2-dihydro-pyridin-3-yl)-6-(4-chloro-phenyl)-2-(2,4-dimethoxy-pyrimidin-5-yl)-1-isopropyl-5,6-dihydro-1H-pyrrolo[3,4-d]imidazol-4-one, and a cyclin dependent kinase 4/6 (CDK4/6) inhibitor 7-Cyclopentyl-2-(5-piperazin-1-yl-pyridin-2-ylamino)-7H-pyrrolo[2,3-d]pyrimidine-6-carboxylic acid dimethylamide. In addition, the disclosure relates to a pharmaceutical combination product. The disclosure also relates to corresponding pharmaceutical formulations, uses and treatment methods comprising said mdm2/4 inhibitor or a cyclin dependent kinase 4/6 (CDK4/6) inhibitor.

Abstract: Dry blend powder formulations comprising a pharmaceutical formulation containing tetrahydrobiopterin, and methods of making and using the same, are disclosed herein.

Abstract: The present invention provides for the design and synthesis of halogenated thieno- and pyrrolopyrimidine compounds and prodrugs thereof that exhibit cancer proliferation inhibitory activity and the use thereof for cancer treatment.

Abstract: The present invention relates to pharmaceutical combinations comprising a PPAR agonist and a P38 inhibitor for use in a method for the prevention, delay of progression or treatment of cancer.

Abstract: The present application relates to a pharmaceutical composition containing compound 5-fluoro-3-phenyl-2-[(15)-1-(7H-purin-6-ylamino)propyl]-4-3H)-quinazolinone, and a pharmaceutically acceptable salt thereof for preventing and treating liver cancer, and the composition of the present application inhibits ROS-PI3K-AKT-TERT signal transduction, and thus can be favorably used in the treatment of a liver cell cancer, in which a protein involved in the signal transduction is overexpressed, especially, advanced liver cell cancer.

Abstract: The present invention refers to the use of sodium channel blockers such as tetrodotoxin or saxitoxin, its analogues/derivatives as well as their acceptable salts, for the production of a medicament for the treatment of neuropathic pain resulting from chemotherapy.

Abstract: The present invention relates to the combination of histamine-3 receptor inverse agonist and acetylcholinesterase inhibitor. Also, the present invention provides histamine-3 receptor (H3R) inverse agonist, or the pharmaceutically acceptable salt(s) thereof in combination with or as adjunct to acetylcholinesterase inhibitor and their use in the treatment of cognitive disorders. The present invention further provides the pharmaceutical composition containing the said combination.

Abstract: The current disclosure relates to a novel therapeutic strategy for patients with hypertriglyceridemia and/or chylomicronemia, wherein the patient is determined to have autoantibodies to GPIHBP1. It was unexpectedly found that autoantibodies to GPIHBP1, a GPI anchored protein of capillary endothelial cells that shuttles lipoprotein lipase to its site of action in the capillary lumen, were found to be present in patients with hypertriglyceridemia, and that the autoantibodies blocked the binding of lipoprotein lipase (LPL) to GPIHBP1. Accordingly, aspects of the disclosure relate to a method for treating hypertriglyceridemia and/or chylomicronemia in a patient comprising administering a therapeutically effective amount of an immunosuppressive treatment and/or GPIHBP1 activator to the patient; wherein the patient has been determined to have GPIHBP1 autoantibodies.

Abstract: The present invention provides compositions and methods for providing sustained release of an active agent through the skin of a subject, wherein a pharmaceutical percutaneous composition comprises at least one fatty acid ester and a therapeutically effective amount of active agent.

Abstract: The disclosure provides a method of treating a mammal having a genetic epileptic disorder, comprising chronically administering a pharmaceutically acceptable pregnenolone neurosteroid to a mammal having a genetic epileptic disorder in an amount effective to reduce the seizure frequency in the mammal. In certain preferred embodiments, the mammal is a human patient who has a CDKL5 genetic mutation. In certain preferred embodiments, the patient has a low endogenous level of a neurosteroid(s). In certain preferred embodiments, the pregnenolone neurosteroid is ganaxolone.

Abstract: The present invention is in the field of a use of a bisphophosphonate acid, specifically a salt thereof, or analogue thereof, or combinations thereof, in a medicament for naturally occurring diseases/disorders in a mammal, the mammal being selected from the orders of Perissodactyla, such as the family of Equidae (horses), (Cet) Artiodactyla, such as the family of Bovidae (cattle), and Carnivora, such as the families of Canidae (e.g. dogs) and Felidae (e.g. cats).

Abstract: Oral dosage forms of osteoclast inhibitors, such as neridronic acid, in an acid or a salt form can be used to treat or alleviate pain or related conditions, such as pain associated with complex regional pain syndrome.

Abstract: Methods of treating anti-inflammatory conditions through the use of boron-containing small molecules are disclosed.

Abstract: The present invention relates to a nutritional composition comprising at least one fucosylated oligosaccharide and at least one N-acetylated oligosaccharide for use in preventing and/or treating non-rotavirus diarrhea in infants or young children by acting on the dysbiosis of the microbiota preceding and/or following the non-rotavirus diarrhea. The composition can be an infant formula and is in particular intended for infants between 0 and 12 months of age fed predominantly with infant formula. It promotes a healthy intestinal flora and has beneficial short and long terms effects.

Abstract: The present invention provides compounds of formula (I) that are prodrugs of catecholamine for use in treatment of neurodegenerative diseases and disorders. The present invention also provides pharmaceutical compositions comprising compounds of the invention and methods of treating neurodegenerative or neuropsychiatric diseases and disorders using the compounds of the invention, in particular Parkinson's disease.

Abstract: Compositions and methods are disclosed for treating subjects with cancer, particularly skin cancer.

Abstract: The present invention relates to the use of cyanoglucoside according to formula (I) and formulations thereof in the prevention and/or treatment of diabetes and/or metabolic syndrome; wherein R1, R2 and R3 are independently selected from an optionally substituted mono- or di-saccharide, —O—C(O)—R4 and halogen, such as Cl, F or Br, wherein R4 is selected from H, alkyl, such as methyl, as well as tautomers, geometrical isomers, optically active forms, pharmaceutically acceptable salts and pharmaceutically active derivative thereof.

Abstract: Provided herein are methods for treating a pulmonary infection in a patient in need thereof, for example, a nontuberculous mycobacterial pulmonary infection for at least one treatment cycle. The method comprises administering to the lungs of the patient a pharmaceutical composition comprising a liposomal complexed aminoglycoside comprising a lipid component comprising electrically neutral lipids and an aminoglycoside. Administration comprises aerosolizing the pharmaceutical composition to provide an aerosolized pharmaceutical composition comprising a mixture of free aminoglycoside and liposomal complexed aminoglycoside, and administering the aerosolized pharmaceutical composition via a nebulizer to the lungs of the patient. The methods provided herein result in a change from baseline on the semi-quantitative scale for mycobacterial culture for a treated patient, and/or NTM culture conversion to negative during or after the administration period.

Abstract: Provided, among other things, is a method of treating or ameliorating pulmonary infection in a cystic fibrosis patient comprising pulmonary administration of an effective amount of a liposomal/complexed antiinfective to the patient, wherein the (i) administrated amount is 50% or less of the comparative free drug amount, or (ii) the dosing is once a day or less, or (iii) both.

Abstract: The present invention includes Uttroside B compositions and method for the treatment of hepatocellular carcinoma. Chemotherapeutic options for liver cancer are limited and the prognosis of HCC patients remains dismal. Sorafenib, is the only drug currently available for the treatment of hepatocellular carcinoma.

Abstract: The present invention provides a method of treating a cancer in a subject comprising administering to the subject an effective amount of a CD33-targeted antibody-drug conjugate (ADC) and an effective amount of cytarabine. Also provided are pharmaceutical compositions comprising an effective amount of a CD33-targeted ADC and an effective amount of cytarabine.

Abstract: Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating AGT and modulating a RAAS pathway related disease, disorder and/or condition in an individual in need thereof. A RAAS pathway related disease, disorder and/or condition in an individual such as hypertension can be treated, ameliorated, delayed or prevented with the administration of antisense compounds targeted to AGT.

Abstract: The present invention relates to small interfering RNA (siRNA) molecules against the HTT gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating Huntington's Disease (HD) using the siRNA molecules and AAV vectors.

Abstract: A hemostatic tissue sealant sponge and a spray for acute wounds are disclosed. The sponge comprises hydrophobically modified polymers that anchor themselves within the membrane of cells in the vicinity of the wound. The seal is strong enough to substantially prevent the loss of blood inside the boundaries of the sponge, yet weak enough to substantially prevent damage to newly formed tissue upon recovery and subsequent removal of the sponge. In examples, the polymers inherently prevent microbial infections and are suitable for oxygen transfer required during normal wound metabolism. The spray comprises hydrophobically modified polymers that form solid gel networks with blood cells to create a physical clotting mechanism to prevent loss of blood. In an example, the spray further comprises at least one reagent that increases the mechanical integrity of the clot. In another example, the reagent prevents microbial infection of the wound.

Abstract: Provided is an inhalation gas device for a therapy of a disease accompanied by epileptiform discharges comprising a medical gas bottle, and a medical inhalation gas device connected to the medical gas bottle, the inhalation gas device for a therapy of a disease accompanied by epileptiform discharges including the following 1) and 2): 1) a therapeutic agent for a disease accompanied by epileptiform discharges containing carbon dioxide as an active ingredient being filled in the medical gas bottle; and 2) the medical inhalation gas device being provided with a gas inhalation mask.

Abstract: Described herein is a method for eliminating or reducing ototoxicity in patients receiving a platinum based chemotherapeutic. In particular, are methods of reducing ototoxicity in a pediatric patient. The methods described herein include administering an effective amount of sodium thiosulfate to a patient in need thereof to reduce ototoxicity.