Abstract: Disclosed are methods for treating a meylodysplastic syndrome (MDS) in a subject that involves administering to the subject a therapeutically effective amount of an inflammasome inhibitor. Also disclosed are methods for diagnosing a myelodysplastic syndrome (MDS) in a subject. In some embodiments, the method involves assaying a sample from the subject to detect inflammasome activation, wherein an increase in inflammasome activation in the sample compared to a control is an indication of MDS in the subject. In some embodiments, the method involves assaying a sample from the subject to detect s100A9 protein levels, wherein an increase in s100A9 protein levels in the sample compared to a control is an indication of MDS in the subject. The disclosed methods can further involve treating the subject for MDS if an increase in inflammasome activation and/or s100A9 levels are detected.

Abstract: The present invention is directed to a method for inhibiting growth of ovarian cancer cells in a subject in need thereof, comprising administering to said subject a composition comprising an effective amount of 4-acetyl-antroquinonol B or a pharmaceutical acceptable salt thereof, and a pharmaceutically acceptable carrier.

Abstract: Compounds, methods, and compositions are provided for the treatment of cancer, neurological disorders, and fibrotic disorders. Specifically, the invention includes administering an effective amount of a compound of Formula I, II, or III, or a pharmaceutically acceptable composition, salt, isotopic analog, prodrug, or combination thereof, to a subject suffering from a cancer, neurological disorder, or fibrotic disorder.

Abstract: Provided is a novel medicament capable of reducing the side effects of metformin or a pharmaceutically acceptable salt thereof and useful as an anti-malignant tumor agent, in which metformin or a pharmaceutically acceptable salt thereof and di-hydroquercetin or a pharmaceutically acceptable salt thereof are combined.

Abstract: A pharmaceutical composition is prepared from the following raw materials (in parts by weight): silybin (8.75-60), phospholipid (15-65), a Pu'er tea extract (25-200), and vitamin E (6.25-40). The composition has the effect of treating non-alcoholic fatty liver diseases.

Abstract: The present disclosure is directed to compositions comprising bryostatin-1, and methods comprising administering a composition comprising bryostatin-1, to treat Niemann-Pick Type C in a subject in need thereof.

Abstract: A kit for treating psychosis by administering a pharmaceutically acceptable liquid composition of Asenapine to oral mucosa includes a formulation having Asenapine, or a pharmaceutically acceptable salt or hydrate thereof, ethanol, and water and a multidose pump filled with the formulation. The Asenapine compositions are stable for an extended period of time at normal storage conditions.

Abstract: A combination of a non-selective, peripheral anticholinergic agent, and a muscarinic receptor agonist, optionally with an acetyl cholinesterase inhibitor, and method of using the same for the treatment of hypocholinergic disorders of the central nervous system. The combination of the present invention allows for safe administration of high doses of muscarinic receptor agonist, and improved efficacy of the muscarinic receptor agonist for treatment of hypocholinergic disorders of the central nervous system. The combination also allows for a maximum supply of acetylcholine to the central nervous system, when an acetyl cholinesterase inhibitor is used in combination with a non-selective, peripheral anticholinergic agent and a muscarinic receptor agonist.

Abstract: Compositions comprising melatonin or derivatives thereof are provided for topical administration to the vaginal and/or rectal epithelium to protect against vaginal and/or rectal radiation injury due to radiotherapy.

Abstract: The present invention is directed to methods of treating and/or ameliorating portal hypertension by administration of a therapeutically effective amount of ifetroban or a pharmaceutically acceptable salt thereof.

Abstract: There is provided novel small molecule E3 ubiquitin ligase protein binding ligand compounds, having utility in PROteolysis Targeted Chimeras (PROTACs), as well as processes for the preparation thereof, and use in medicine. There is particularly provided PROTACs which bind to a protein within the bromo- and Extra-terminal (BET) family of proteins, and especially to PROTACs including novel small molecule E3 ubiquitin ligase protein binding ligand compounds which selectively induce degradation of the BRD4 protein within the bromodomain of the BET family of proteins.

Abstract: The present invention provides a compound of Formula I and pharmaceutical compositions comprising one or more said compounds, and methods for using said compounds for treating or preventing thromboses, embolisms, hypercoagulability or fibrotic changes. The compounds are selective Factor XIa inhibitors or dual inhibitors of Factor XIa and plasma kallikrein.

Abstract: Transdermal therapeutic system and method of using the same for safely treating hypocholinergic disorders of the central nervous system such as Alzheimer type dementia. The transdermal therapeutic system comprises oxybutynin in combination with a cholinergic receptor agonist (CRA) such as xanomeline.

Abstract: The invention relates to compounds of formula I: where a, R1, and R3-6 are as defined in the specification, or a pharmaceutically acceptable salt thereof. The compounds of formula I are serotonin and norepinephrine reuptake inhibitors. The invention also relates to pharmaceutical compositions comprising such compounds; methods of using such compounds; and process and intermediates for preparing such compounds.

Abstract: This invention is directed to derivatives of piperidine-2,6-dione, or their organic or inorganic salts thereof, a methods of synthesis of these derivatives, and their application as active pharmaceutical ingridient as inhibitors of TNF? releasing in cells, the derivative of piperidine-2,6-dione being of the general formula (I): wherein n represents 1, 2, 3, 4, 5 or 6; R1 represents from one to four of the same or different substituents selected from F, Cl, Br, C1-4 alkyl, OH, OC1-4 alkyl, NO2, NHC(O)C1-4 alkyl, NH2, NH(C1-4 alkyl), N(C1-4 alkyl)2; R2 represents OR3, NR3R4, N(R3)COR4, O2CR5; R3 and R4 represent independently and at each occurrence H or C1-4 alkyl; R5 represents CHR6NR7R8, CHR6NR9C(O)CHR10NR7R8, a heterocycle W or CHR6NR9C(O)W; R6, R9, R10 represent independently and at each occurrence H, or C1-4 alkyl; R7 and R8 represent independently and at each occurrence H, C14 alkyl, or R7 and R8 taken together represent 1,3-propylene, 1,4-butylene, 1,5-pentylene , or 1,6-hexylene; W represents four-m

Abstract: Ophthalmic compositions and methods of preparing such compositions are disclosed.

Abstract: The present disclosure provides methods of treating with a BTK inhibitor a blistering disease, in particular pemphigus vulgaris or pemphigus folliaceous in a mammal, use of a BTK inhibitor as a replacement therapy for corticosteroid therapy for diseases treatable with a corticosteroid, such as autoimmune or inflammatory disease and in particular where corticosteroids are used as first or second line therapy, and pharmaceutical formulations comprising the same.

Abstract: The invention provides a solid composition of the peripheral mu opioid antagonist axelopran and a combination dosage form of the mu opioid antagonist axelopran sulfate in an immediate release form and an opioid analgesic agent which may be in an extended release, sustained release, modified release, or controlled release form and methods of preparing such a combination dosage form.

Abstract: The present invention provides connexin hemichannel blockers for the treatment of epilepsy. In particular, the present invention relates to organic compounds which act as selective blockers connexin hemichannels which are useful in the treatment of epilepsy. In another embodiment of the present invention there is provided a composition comprising a selective connexin hemichannels blocker compounds useful in the treatment of epilepsy in combination with other antiepileptic compounds.

Abstract: The present invention concerns a combination comprising tasquinimod, or a pharmaceutically acceptable salt thereof, and a PD-1 and/or PD-L1 inhibitor, for use as a medicament. It also concerns a pharmaceutical composition comprising tasquinimod, or a pharmaceutically acceptable salt thereof, and PD-1 and/or PD-L1 inhibitor.

Abstract: This invention provides a method of treating a subject afflicted with multiple sclerosis or presenting a clinically isolated syndrome comprising administering to the subject laquinimod as an add-on therapy to or in combination with DMF. This invention also provides a package comprising laquinimod and DMF for treating a subject afflicted with multiple sclerosis or presenting a clinically isolated syndrome. This invention also provides laquinimod for use as an add-on therapy or in combination with DMF in treating a subject afflicted with multiple sclerosis or presenting a clinically isolated syndrome. This invention also provides a pharmaceutical composition comprising laquinimod and DMF for use in treating a subject afflicted with multiple sclerosis or presenting a clinically isolated syndrome. This invention further provides use of laquinimod and DMF in the preparation of a combination for treating a subject afflicted with multiple sclerosis or presenting a clinically isolated syndrome.

Abstract: The present invention relates to a solid pharmaceutical composition of temozolomide that has good and consistent flowability as a powder and taste masking and is readily dispersible in an aqueous solution suitable for oral administration, e.g., as a dry sprinkle. This permits patients and healthcare workers to accurately measure doses and safely dispense the drug.

Abstract: Methods of treating disorders with a combination of antifungals is provided. The methods include topically administering a combination of antifungal agents to an affected tissue are provided. For example, the ostiomeatal complex of a subject having a recurring condition can receive the formulations taught herein, the topically administering including contacting a first antifungal agent with the ostiomeatal complex, the first antifungal selected as effective at killing Candida species of fungus, inhibiting the growth and/or reproduction of the Candida species, or a combination thereof; and, contacting a second antifungal agent with the ostiomeatal complex, the second antifungal selected as effective at killing an Aspergillus species of fungus, inhibiting the growth and/or reproduction of the Aspergillus species, or a combination thereof; wherein, the topically administering of the first antifungal agent is concurrent with the topically administering of the second antifungal agent.

Abstract: Methods of treating disorders with a combination of antifungals is provided. The methods include topically administering a combination of antifungal agents to an affected tissue are provided. For example, the ostiomeatal complex of a subject having a recurring condition can receive the formulations taught herein, the topically administering including contacting a first antifungal agent with the ostiomeatal complex, the first antifungal selected as effective at killing Candida species of fungus, inhibiting the growth and/or reproduction of the Candida species, or a combination thereof; and, contacting a second antifungal agent with the ostiomeatal complex, the second antifungal selected as effective at killing an Aspergillus species of fungus, inhibiting the growth and/or reproduction of the Aspergillus species, or a combination thereof; wherein, the topically administering of the first antifungal agent is concurrent with the topically administering of the second antifungal agent.

Abstract: Methods of treating disorders with a combination of antifungals is provided. The methods include topically administering a combination of antifungal agents to an affected tissue are provided. For example, the ostiomeatal complex of a subject having a recurring condition can receive the formulations taught herein, the topically administering including contacting a first antifungal agent with the ostiomeatal complex, the first antifungal selected as effective at killing Candida species of fungus, inhibiting the growth and/or reproduction of the Candida species, or a combination thereof; and, contacting a second antifungal agent with the ostiomeatal complex, the second antifungal selected as effective at killing an Aspergillus species of fungus, inhibiting the growth and/or reproduction of the Aspergillus species, or a combination thereof; wherein, the topically administering of the first antifungal agent is concurrent with the topically administering of the second antifungal agent.

Abstract: The present invention relates to a therapeutic agent for chronic myelogenous leukemia with reduced drug-resistance and side-effects containing a 1,6-disubstituted indole compound.

Abstract: A compound for treating a disease, in particular cancer like non-small cell lung cancer, exceptionally inhibits activity of oncogenic ALK kinase. Compositions, particularly pharmaceutical compositions, are provided comprising this compound. Methods for targeting cancer cells harboring an abnormality in the ALK gene are also The compound for treating a disease has certain structural elements, namely a tricyclic, more specifically heterocyclic, backbone as the core part of the compound at least one highly electronegative atom in form of a tertiary amine attached to the backbone via an at most 6-membered linking group with a terminal highly electronegative atom in form of a nitrogen as secondary amine, and a further hydrophobic moiety fused to the backbone. The structural components allow for an advantageous interaction with the ALK kinase domain. The compound therefor represents a highly promising treatment option for patients in particular those bearing ALK-dependent non-small cell lung cancer.

Abstract: Disclosed herein are methods of treating diseases that exhibit over-activation of the ERBB4 pathway, such as breast cancer and lung cancer. The method comprises administering to a subject having a disease that exhibits over-activation of the ERBB4 pathway and a gene expression profile consistent with an ERBB4-sensitive profile a therapeutically effective amount of an ERBB4 inhibitor, wherein the disease is effectively treated. Gene expression profiles of ERBB4-sensitive cells and microarrays suitable for protein-tyrosine kinases are also provided.

Abstract: Methods and compositions for enhancing the ability of hypoxia-activated bioreductive agents to kill tumor cells within solid tumors are provided. Local regions of hypoxia are created within a tumor, or within a region containing a tumor, resulting in enhanced activation of hypoxia-activated bioreductive agents (e.g. tirapazamine) within the local region. The activated hypoxia-activated bioreductive agents kill tumor cells in the hypoxic region by to catalyzing DNA stand breakage within the tumor cells. Because the activity is localized, side effects that typically occur as a result of systemic administration of bioreductive agents are reduced.

Abstract: The present invention relates to 4-(4-Fluoro-2-methoxyphenyl)-N-{3-[(S-methylsulfonimidoyl)methyl]phenyl}-1,3,5-triazin-2-amine (compound A), more particularly (+)-4-(4-Fluoro-2-methoxyphenyl)-N-{3-[(S-methylsulfonimidoyl)methyl]phenyl}-1,3,5-triazin-2-amine (compound A?), for use in treating lymphoma, preferably diffuse large B-cell lymphoma (DLBCL) mantle cell lymphoma, follicular lymphoma, diffuse large B-cell lymBurkitt's lymphoma, adult T-cell lymphoma (ATL) and Hodgkin's lymphoma, more preferably DLBCL and ATL.

Abstract: The present invention relates to the use of PIKfyve inhibitors to treat Niemann-Pick disease type C, and related compositions and methods.

Abstract: Unit dosage forms of meloxicam containing either 5 mg or 10 mg of meloxicam that provide effective pain relief and have desirable pharmacokinetic properties are described. The unit dosage forms can provide pain relief when a single unit dose is administered to a patient and useful for treating pain such as osteoarthritis pain at a relatively low systemic exposure to meloxicam.

Abstract: The use of BETi as a potential treatment for FSHD is provided. Specifically, the use of BETi, and particularly selective BETi for BRD4, are shown to inhibit DUX4 expression which is expected to result in a decrease in the severity of symptoms of FSHD. Further, the treatments are shown to work when pulsed as opposed to continuous. This allows for a BETi to be supplied to a human in a pulse, and then allows the human to not need any additional treatment for a window at least as long as the one of the treatment pulse.

Abstract: A method for treating a subject suffering from a cancer, in particular a multidrug-resistant cancer includes administrating a cobalt-polypyridyl complex to the subject. A method for suppressing the growth of cancer cells, in particular inducing autophagy of the cancer cells, inducing cell cycle arrest of the cancer cells and/or inhibiting cell invasion of the cancer cells and for specifically targeting cancer cells with multidrug-resistance includes contacting said cancer cells with the cobalt-polypyridyl complex. A pharmaceutical composition and a kit are provided and include the cobalt-polypyridyl complex. Unexpectedly, the cobalt-polypyridyl complex is especially suitable to treat cancer, in particular multidrug-resistant cancer with an exceptionally increased cytotoxic activity towards multidrug-resistant cancer cells.

Abstract: A method for inhibiting the expression and/or functional activity of an ABC transporter protein in a subject suffering from a disorder associated with an overexpression of the ABC transporter protein includes administrating a cobalt-polypyridyl complex to the subject. A method for inhibiting the expression and/or functional activity of an ABC transporter in cells includes contacting the cells with an effective amount of the cobalt-polypyridyl complex.

Abstract: The invention provides a fulvestrant composition comprising not more than 2% total impurities that comprises a nonionic surfactant and/or a ricinoleate vehicle and which, optionally, is free of a non-aqueous ester solvent. The composition may be used to treat hormone receptor positive metastatic breast cancer in a subject.

Abstract: The present invention provides cortexolone 17?-propionate for use in the treatment of skin wounds and/or atrophic skin disorders. The present invention also provides pharmaceutical or cosmetic compositions comprising cortexolone 17?-propionate for use in the treatment of skin wounds and/or of atrophic skin disorders.

Abstract: The invention provides novel conjugate compounds having at least one of a moiety derived from ursodeoxycholic acid, or eicosapentaenoic acid, or docosahexaenoic acid, or rhein, or R-(+)-?-lipoic acid, or ursolic acid, or corosolic acid, or hydroxycitric acid, or cinnamic acid, or cholic acid, or oleanolic acid, or salicylic acid, or betulinic acid, or chlorogenic acid, or caffeic acid, or bassic acid, or acetyl L-carnitine, or S-allyl cysteine sulphoxide, or S-methyl cysteine sulfoxide, or pantothenic acid, or ascorbic acid, or retinoic acid, or nicotinic acid, or biotin, or a derivative or analog thereof, and a moiety derived from berberine or L-carnitine or metformin or unsaturated fatty acid, or a derivative or analog thereof.

Abstract: The compositions and methods disclosed herein provide for treatments of ocular disorders of the eye, such as ocular surface diseases as well as those associated with inflammation of internal cells and cell layers of the eye. The methods disclosed herein describe methods for treating ocular disorders, particularly dry eye disorders, comprising topically administering to an ocular region of a subject a composition comprising a pharmaceutically effective amount of at least one aldosterone antagonist or salt thereof and a pharmaceutically acceptable carrier. Included is a method for treating dry eye comprising topically administering to an ocular region of a subject a composition comprising spironolactone and hydroxypropyl methylcellulose; and reducing or preventing one or more symptoms or causes of dry eye.

Abstract: In one aspect, this disclosure describes a pharmaceutical composition that generally includes frankiamicin A and a pharmaceutically acceptable carrier. In another aspect, this disclosure describes a method of treating a subject having, or at risk of having, a condition caused by a microbial infection treatable with frankiamicin A. Generally, the method includes administering to the subject an amount of frankiamicin A effective to ameliorate at least one symptom or clinical sign of the condition.

Abstract: Disclosed are compounds of Formula 1, stereoisomers thereof and pharmaceutically acceptable salts of the compounds and stereoisomers, wherein R1 and R2 are defined in the specification. This disclosure also relates to materials and methods for preparing compounds of Formula 1, to pharmaceutical compositions which contain them, and to their use for treating obesity and related diseases, disorders, and conditions associated with MetAP2.

Abstract: In one aspect, the invention relates to compounds having the formula: where R1-R6, a, b, Z, and X are as defined in the specification, or a pharmaceutically acceptable salt thereof. These compounds have neprilysin inhibition activity. In another aspect, the invention relates to pharmaceutical compositions comprising such compounds; methods of using such compounds; and processes and intermediates for preparing such compounds.

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

Abstract: Disclosed are methods and compositions related to methods of treating, ameliorating, mitigating, slowing, arresting, preventing or reversing various diseases and conditions, including age-related obesity, age-related increases in blood lipid levels, age-related decreases in insulin sensitivity, age-related decreases in memory function, and age-related changes in eye function such as macular degeneration. The methods comprise administering nicotinamide mononucleotide (NMN) to a subject. In some embodiments, the administration can be oral administration. Also disclosed are pharmaceutical compositions comprising NMN.

Abstract: Drug delivery to the cancer cells during its treatment is challenging. Most of the cancer drug cannot reach to the target because of its complex membrane physiology, drug resistance, and mutability. Establishment of a potential target molecule or domain on the cancer cell is the rate limiting step for the successful drug delivery. Cancer is characterized by abnormal energy metabolism shaped by nutrient deprivation that malignant cells experience during various stages of tumor development. This patent demonstrated a new method of establishing a new target domain that could have enormous importance for the drug delivery during cancer treatment. The inventors showed how nutrient-deprived cancer cells become involved in robust membrane glycan display while treating with nucleotide sugar such as sialic acid and that glycosylated membrane domain become a target domain of cancer cell treatment.

Abstract: Provided are a conjugate including a core and sialic acids or derivatives thereof bound to the surface of the core, and use thereof. The conjugate provided in the present invention binds with hemagglutinin on the surface of influenza virus to inhibit the course of infection of influenza virus, thereby preventing or treating infection of influenza virus and also preventing or treating infection of influenza virus resistant to antiviral agents. Accordingly, the conjugate may be widely used in the development of prophylactic or therapeutic agents for influenza virus infection.

Abstract: Disclosed are a crystallization water-free calcium dibutyryladenosine cyclophosphate crystal form, and a preparation method and a use thereof. In an X-ray powder diffraction pattern using Cu-K? as a source of radiation, the crystallization water-free calcium dibutyryladenosine cyclophosphate crystal form has characteristic peaks at positions where diffraction angles 2? are equal to 12.3°±0.2°, 17.6°±0.2°, 21.4°±0.2°, 24.7°±0.2°, 25.3°±0.2° and 27.8°±0.2°. The crystallization water-free calcium dibutyryladenosine cyclophosphate crystal form of the present invention has a high purity, and good stability; the preparation method is simple and convenient, has good reproducibility, and is easy to industrially popularize and apply.

Abstract: This invention relates to a nucleic acid aptamer specifically binding to avian influenza virus subtype H5 and a method of detecting avian influenza virus subtype H5 using the same, and more particularly to a method of detecting avian influenza virus subtype H5, which is able to rapidly check the presence and concentration of avian influenza virus subtype H5 using a nucleic acid aptamer specifically binding to hemagglutinin, which is a surface protein of avian influenza virus subtype H5.

Abstract: RNA encoding an immunogen is co-delivered to non-immune cells at the site of delivery and also to immune cells which infiltrate the site of delivery. The responses of these two cell types to the same delivered RNA lead to two different effects, which interact to produce a strong immune response against the immunogen. The non-immune cells translate the RNA and express the immunogen. Infiltrating immune cells respond to the RNA by expressing type I interferons and pro-inflammatory cytokines which produce a local adjuvant effect which acts on the immunogen-expressing non-immune cells to upregulate major histocompatibility complex expression, thereby increasing presentation of the translated protein to T cells. The effects on the immune and non-immune cells can be achieved by a single delivery of a single RNA e.g. by a single injection.

Abstract: The invention provides a method of treating preeclampsia, fetal growth restriction (FGR or IUGR), obesity in pregnancy, post-partum cardiomyopathy (heart failure in mothers), cancer and diabetic retinopathy, cardiomyopathy, myocardial infarction, wet microdegeneration and other disorders where angiogenesis is aberrant either diminished or exacerbated, comprising modulating the activity of micro RNA (miRNA). Also provided are an MiRNA modulator or a combination thereof or functional fragments or homologues thereof for use in the treatment of preeclampsia, fetal growth restriction, obesity in pregnancy, cancer, and diabetic retinopathy, cardiomyocyte infarction, wet microdegeneration as well as other disorder where angiogenesis is aberrant either diminished or exacerbated. Methods of modulating angiogenesis and pharmaceutical compositions using miR-122, miR-374b or inhibitors of miR-152 or miR195 are also provided together with methods of diagnosis using the miRNAs.