Abstract: Compositions and methods for the treatment of Charcot-Marie-Tooth disease and related disorders. Also provided are combination therapies for treating this disease by decreasing PMP22 expression in a subject.

Abstract: The present invention relates to the use of a 2,5-dihydroxybenzene derivative represented by Formula (I) or a pharmaceutically acceptable salt, solvate, isomer, or prodrug thereof for the therapeutic and/or prophylactic treatment of, inter alia, dermatitis.

Abstract: The present invention relates to a composition appropriate for topical application, containing fusidic acid. The homogeneity of the fusidic acid is 90% to 110% of the suggested fusidic acid content, and the amount of air present in the cream is lower than 5 vol %. Furthermore, the present invention also relates to a method for preparing the composition. First, a placebo cream and a suspension of fusidic acid are prepared, which are subsequently mixed at a temperature above the melting point of the placebo cream.

Abstract: Provided are compounds of formula (I), wherein: ring A and ring B are each independently an optionally substituted 5-6 membered monocyclic aryl or heteroaryl; one of X, Y and W is CH and the two others are N; and Z is H or —C(R1)(R2)(R3). The compounds are inhibitors of isocitrate dehydronenase 2 (IDH2) mutants useful for treating cancer.

Abstract: The present invention relates to compounds of formula (I) and salts thereof, pharmaceutical compositions containing such compounds and to their use in therapy

Abstract: The present invention relates to a method of treating motor neuron diseases such as amyotrophic lateral sclerosis (ALS) using levosimendan or its active metabolite (II) as an active ingredient. Levosimendan or its active metabolite (II) are able to relieve the loss of skeletal muscle strength or function associated with motor neuron diseases.

Abstract: The subject invention provides therapeutic compositions and uses thereof for improving pulmonary function. In one embodiment, the therapeutic composition comprises one or more free amino acids selected from lysine, glycine, threonine, valine, tyrosine, aspartic acid, isoleucine, tryptophan, asparagine, and serine; and electrolytes. In one embodiment, the subject invention can be used to prevent or treat long-term lung complications induced by radiation.

Abstract: The present invention is directed to compositions and methods for delivering a tissue scaffold comprising ultrasound-triggerable agents to an individual.

Abstract: The present invention relates to the use of one or more compounds selected from the following: caffeic acid, thymol, aspirin, benzydamine hydrochloride, diclofenac sodium, flurbiprofen, ibuprofen, indomethacin, trifluoperazine hydrochloride, chlorprothixene hydrochloride, triflupromazine hydrochloride, suloctidil, thioridazine hydrochloride, dichlorophen, saccharin and piroxicam, in combination with a polymyxin selected from colistin or polymyxin B or a pharmaceutically acceptable derivative thereof, for use in the treatment of a microbial infection, and in particular for killing clinically latent microorganisms associated with microbial infections. The invention also provides a combination comprising suloctidil or a pharmaceutically acceptable derivative or prodrug thereof, and a polymyxin selected from polymyxin E and polymyxin B or a pharmaceutically acceptable derivative thereof. This combination is particularly useful for the treatment and/or prevention of microbial infections.

Abstract: The present invention relates to a process for the preparation of compounds endowed with phosphodiesterase (PDE4) inhibitory activity having formula (I). The invention also relates to the process for the isolation by crystallization of the compound (I) and to its use for the preparation of pharmaceutical compositions for inhalation in combination with suitable carriers or vehicles. The present invention also relates to solvates and crystal forms of a compound of formula (I). The synthesized product is suitable for use in pharmaceutical applications for instance in the treatment of respiratory diseases.

Abstract: Compositions and methods for the treatment of Charcot-Marie-Tooth disease and related disorders. Also provided are combination therapies for treating this disease by decreasing PMP22 expression in a subject.

Abstract: The present disclosure is directed to methods, compositions, and kits for treating dementia.

Abstract: A method of treating steroid-dependent disease such as prostate cancer in a subject is described that includes administering a therapeutically effective amount a CYP17A inhibitor and an effective amount of a 5-?-reductase inhibitor to the subject.

Abstract: A method for preparing functional edible oil rich in phytosterol esters and diglycerides includes steps of: 1) adding a raw material: adding phytosterol, triglyceride and a molecular sieve into a reactor, wherein a ratio of the phytosterol and the triglyceride is 1:2-1:4, a molecular sieve amount is 50 g/L; heating to 50-60° C. and stirring for 30-60 min, for obtaining a pre-mixture; 2) providing non-aqueous enzymatic transesterification: adding 5-20 g/L lipase into the pre-mixture, adding 100-200 ppm antioxidant, stirring and reacting for 8-12 h with a temperature of 50-60° C. and an atmospheric pressure, stopping heating and naturally cooling to a room temperature; and 3) post-treating: after reaction, removing the lipase and the molecular sieve by centrifugation, for obtaining the functional edible oil. The functional edible oil rich in two nutritional active components is obtained by the one-step method. Products of the present invention do not need separation and purification, and operation is simple.

Abstract: Provided herein are methods of utilizing bile acid transport inhibitors and/or enteroendocrine peptide enhancing agents for the treatment of obesity, diabetes, and inflammatory gastrointestinal conditions.

Abstract: A transmembrane penetration enhancer includes a thermotropic liquid crystal suitably prepared for use in a therapeutic system to improve delivery of at least one active ingredient across a biomembrane and/or skin. The thermotropic liquid crystal comprises at least one cholesteryl ester and is effective in a mesomorphic state at a temperature of the biomembrane and/or skin. A therapeutic system includes the prepared thermotropic liquid crystal for enhanced transmembrane penetration and at least one active ingredient for delivery by the thermotropic liquid crystal. A method for making and using the transmembrane penetration enhancer is presented.

Abstract: Modulators of adenosin A3 receptors of formula (I): And procedure for preparing these compounds. Other objectives of the present invention are to provide pharmaceutical compositions comprising an effective amount of these compounds and the use of the compounds for manufacturing a medicament for the treatment of pathological conditions or diseases that can improve by modulation of the adenosine A3 receptor.

Abstract: Certain embodiments of the invention relates to a transdermal drug delivery system containing donepezil or a pharmaceutically acceptable salt thereof as an active ingredient, more specifically to a transdermal drug delivery system containing a drug-containing matrix layer comprising donepezil or its pharmaceutically acceptable salt, a styrene copolymer, a hydrogenated rosin glycerol ester, and optionally a hydrocarbon resin.

Abstract: The present disclosure is directed to generally methods for treating mammographic breast density and/or breast stiffness in a patient in need thereof, such as a premenopausal or perimenopausal patient, comprising the administration of an effective amount of androgenic agent and an effective amount of an aromatase inhibitor. These methods may also be useful in post-menopausal woman.

Abstract: The subject matter generally relates to compounds and methods of treatment and/or prophylaxis of CNS diseases, disorders, and/or injuries. In one aspect, the subject matter relates to inhibitors of phosphodiesterase 1 (PDE1) as neuroprotective agents and/or neural regenerative agents. In a further aspect, the subject matter relates to individuals that are at risk for the development of CNS disease or disorder.

Abstract: The invention relates to a derivative of 15-oxospiramilacton, particularly to a compound of Formula I or II, or an isomer, a solvate or a pharmaceutically acceptable salt thereof. The invention also relates to a pharmaceutical composition comprising the compound as pharmaceutically active ingredient, a method for preparing the same, and use thereof in manufacture of an anti-tumor agent. The derivative of 15-oxospiramilactone of the invention have an activity against multiple tumor cell lines, and the anti-tumor activity is positively correlated to an activity inhibiting the Wnt signaling pathway.

Abstract: The present invention relates to long acting pharmaceutical compositions or pharmaceutically acceptable salts thereof, useful in the treatment or prevention of Human Immunodeficiency Virus (HIV) infections.

Abstract: The present inventor has found out that the following criteria enable to ensure an effect for increasing the platelet count while preventing an excessive increase in the platelet count; “when the platelet count has increased by a certain amount and reached to a sufficient level of the platelet count during administration of a pharmaceutical composition containing a compound having a thrombopoietin receptor agonistic activity, administration of the pharmaceutical composition is discontinued thereafter”.

Abstract: A method of stimulating ventilatory and/or respiratory drive in a subject in need thereof includes administering to the subject a therapeutically effective amount of a composition comprising a cystine ester or a pharmaceutically acceptable salt thereof. Embodiments described herein relate to compositions and methods of stimulating ventilatory and/or respiratory drive in a subject in need thereof, and particularly relates to compositions and methods of treating breathing diseases and/or disorders associated with impaired ventilatory and/or respiratory drive.

Abstract: The present invention describes novel methods for using Treprostinil or its derivative, or a pharmaceutically acceptable salt thereof, for the treatment and/or prevention of ischemic lesions, such as digital ulcers, in subjects with scleroderma (including systemic sclerosis), Buerger's disease, Raynaud's disease, Raynaud's phenomenon and/or other conditions that cause such lesions. The invention also relates to kits for treatment and/or prevention of ischemic lesions, comprising an effective amount of Treprostinil or its derivative, or a pharmaceutically acceptable salt thereof.

Abstract: Embodiments of the invention are directed to methods of treating cancer cells, particularly breast cancer cells, such as chemo-resistant cells, with an Hsp90 inhibitor and an anticancer agent or compound such as chemotherapy.

Abstract: A method for analyzing an aliphatic compound by mass spectrometry which comprises: (i) ionizing an aliphatic compound in the presence of a heterocyclic modifier; and (ii) mass analyzing the resulting ions to obtain mass spectrometric data.

Abstract: Disclosed herein is a method for increasing the dendritic spine formation or dendritic spine density in a subject, who is affected by a dendritic spine defect caused by the impairment in neurofibromin (NF1 protein), valosin-containing protein (VCP), atlastin-1 (ATL1), or superoxide dismutase 1 (SOD1). Accordingly, also disclosed herein is a method for treating a subject having or suspected of having a synaptopathy caused by the impairment in NF1, VCP, ATL1, or SOD1.

Abstract: Disclosed are bicyclic aryl compounds of formula (I), that can modulate the activity of the autotaxin (ATX) enzyme. This invention further relates to compounds that are ATX inhibitors, and methods of making and using such compounds in the treatment of demyelination due to injury or disease, as well as for treating proliferative disorders such as cancer.

Abstract: Methods of diagnosing and treating disorders related to TH2 inhibition, including but not limited to asthma, are provided. Also provided are methods of selecting or identifying patients for treatment with certain therapeutic agents that are TH2 pathway inhibitors.

Abstract: Certain embodiments are directed to methods of identifying a subject having a higher risk of prostate cancer recurrence. The methods can include the step of measuring levels of one or more of FLIP, transcription factor Sp1, and transcription factor Sp3 in a prostate sample from the subject, wherein elevated levels of FLIP, transcription factor Sp1, and transcription factor Sp3 identify a subject as high risk for prostate cancer recurrence.

Abstract: The invention provides methods and materials related to a gene expression-based survival predictor for DLBCL patients.

Abstract: Methods are provided herein for the use of Gli-1 as a specific marker for myofibroblast progenitor cells, thereby permitting the diagnosis of early stages of fibrosis prior to the onset of organ failure. Also provided herein are methods of isolating Gli-1+ cells for e.g., use in high-throughput screening of compounds for the treatment or prevention of fibrosis. In another aspect, provided herein are methods of treating or preventing fibrosis (e.g., kidney fibrosis) in a subject by administering a Gli inhibitor, particularly a Gli-2 inhibitor or a Gli-1/Gli-2 inhibitor.

Abstract: One aspect of the invention relates to AI-helper compositions comprising an AI compound (e.g., melatonin, alpha-lipoic acid, vitamin E, vitamin D, glutathione, resveratrol, astaxanthin, beta carotene, vitamin A, vitamin C, vitamin B12, vitamin B6, folic acid, and taurine) and one or more helper esters. The AI-helper compositions disclosed herein can be used for effective transdermal delivery of the AI compound to a subject. Another aspect of the invention relates to applications and preparations of the AI-helper compositions.

Abstract: The invention relates to compositions and methods useful for enhancing hair growth and promoting skin regeneration. Particularly, the invention provides topical compositions including trimebutine, salts, or active metabolites thereof, for enhancing or inducing hair growth and promoting skin regeneration. Compositions comprising trimebutine or a pharmaceutically acceptable salt or active metabolite thereof and their use in the method of promoting hair growth or skin regeneration. Preferably trimebutine is trimebutine maleate or N-desmethyl trimebutine. Compositions are preferably in a form for topical administration, such as a gel.

Abstract: Disclosed is a levosimendan compound, an optically active isomer thereof, or a pharmaceutically acceptable salt thereof, which is used for the prevention and/or treatment of diseases caused by tau aggregation or phosphorylation, such as neurodegenerative diseases (e.g., Alzheimer disease). In accordance with yet another aspect of the present invention, there is provided a health functional food for preventing or treating tau aggregation-related diseases, containing a levosimendan compound or a pharmaceutically acceptable salt, stereoisomer or solvate thereof.

Abstract: The embodiments described herein include methods and formulations for treating viruses and diseases that are exacerbated by inflammatory responses in the body. The methods and formulations include, but are not limited to, methods and formulations for delivering effective concentrations of levocetirizine and montelukast to a patient in need. The methods and formulations can comprise conventional and/or modified-release elements, providing for drug delivery to the patient.

Abstract: The mixture of un-refined salt and sugar in the manufacture of a medicament employed for treating lax vagina syndrome or colpoxerosis disease in a mammal. Various tests, vaginal smooth muscle contractility test using New Zealand White Rabbit; (Experimental example 1); the effect on the vagina contractility in volunteers by using perinometer (Experimental example 2); and the effect on the colpoxerosis disease in volunteers (Experimental example 3), showed improving effect on the contractility of vagina tissue and colpoxerosis disease. Accordingly, the combination can be useful in treating or preventing lax vagina syndrome or colpoxerosis disease.

Abstract: Crystalline micronized particulate of a glycopyrronium salt may be prepared by hydrodynamic cavitation. The resulting drug particles are physically stable with regard to agglomeration and/or aggregation on storage.

Abstract: Provided is a composition for use in treating a skin condition, the composition comprising: (a) a first component comprising a substituted or unsubstituted diindolylmethane compound; and (b) a second component comprising a substituted or unsubstituted retinoid compound.

Abstract: The mixture of processed salt and sugar in the manufacture of a medicament employed for treating lax vagina syndrome or colpoxerosis disease in a mammal. Various tests, vaginal smooth muscle contractility test using New Zealand White Rabbit; (Experimental example 1); the effect on the vagina contractility in volunteers by using perinometer (Experimental example 2); and the effect on the colpoxerosis disease in volunteers (Experimental example 3), showed improving effect on the contractility of vagina tissue and colpoxerosis disease. Accordingly, the combination can be useful in treating or preventing lax vagina syndrome or colpoxerosis disease.

Abstract: The present disclosure is directed to methods for reducing cardiovascular risk in a human subject by administering atrasentan, or a pharmaceutically acceptable salt thereof, in an amount sufficient to effect a reduction of about 5% or more in one or both of (a) total serum cholesterol, relative to the subject's baseline total serum cholesterol, and (b) serum LDL cholesterol, relative to the subject's baseline serum LDL cholesterol.

Abstract: A use of an inhalable powder formulation for preventing or treating an NMDA receptor hypofunction-related disease, a method for preventing or treating an NMDA receptor hypofunction-related disease in a subject, which comprises administering a therapeutically effective amount of an inhalable powder formulation comprising growth hormone (GH) to the subject in need thereof, and an inhalable powder formulation for preventing or treating an NMDA receptor hypofunction-related disease comprising GH as an active ingredient are provided.

Abstract: Described herein are compounds and pharmaceutical compositions containing such compounds which inhibit cystathionine-?-lyase (CSE). Also described herein are methods for using such CSE inhibitors, alone or in combination with other compounds, for treating diseases or conditions that would benefit from CSE inhibition.

Abstract: A method of analyzing a population of cells is disclosed. In certain embodiments, the method includes i) obtaining an array of cells on a substrate, wherein the cells are labeled with one or more mass tags and are separated from one another, ii) measuring, using secondary ion mass spectrometry (SIMS), the abundance of the one or more mass tags at a plurality of locations occupied by the cells, thereby generating, for each individual cell measured, a set of data, and iii) outputting the set of data for each of the cells analyzed. Also provided herein are systems that find use in performing the subject method. In some embodiments, the system is an automated system for analyzing a population of cells using SIMS.

Abstract: The present invention relates to lower dosage strength pernasal testosterone gel formulations for intranasal administration and treatment methods for using the lower dosage strength pernasal testosterone gel formulations for treating a female subject with anorgasmia and/or hypoactive sexual desire disorder.

Abstract: The invention relates to methods for treating pruritus with NK-1 receptor antagonists such as serlopitant. The invention further relates to pharmaceutical compositions comprising NK-1 receptor antagonists such as serlopitant. In addition, the invention encompasses treatment of a pruritus-associated condition with serlopitant and an additional antipruritic agent, and the use of serlopitant as a sleep aid, optionally in combination with an additional sleep-aiding agent.

Abstract: The present invention relates to respirable dry powder comprising respirable dry particles that comprise sodium chloride, leucine, and tiotropium bromide, wherein the sodium chloride is about 60% to about 90%, the leucine is about 10% to about 40%, the tiotropium bromide is about 0.01% to about 0.5%, and optionally one or more additional therapeutic agents up to about 20%, wherein all percentages are weight percentages on a dry basis and all the components of the respirable dry particles amount to 100%. The invention also relates to respirable dry powders that contain respirable dry particles that comprise sodium chloride, leucine, and tiotropium bromide, wherein the sodium chloride is 67% to 84%, the leucine is 12% to 28%, the tiotropium bromide is about 0.01% to about 0.5%, and optionally one or more additional therapeutic agents up to about 20%, wherein all percentages are weight percentages on a dry basis and all the components of the respirable dry particles amount to 100%.

Abstract: The invention relates to methods for treating pruritus with NK-1 receptor antagonists such as serlopitant. The invention further relates to pharmaceutical compositions comprising NK-1 receptor antagonists such as serlopitant. In addition, the invention encompasses treatment of a pruritus-associated condition with serlopitant and an additional antipruritic agent, and the use of serlopitant as a sleep aid, optionally in combination with an additional sleep-aiding agent.

Abstract: Provided herein is a pharmaceutical composition combination which comprises (a) a corticosteroid and (b) b-adrenergic receptor antagonist characterised in that active ingredients (a) and (b) form part of a single composition. Further provided is a method of treating a human or animal patient suffering from or susceptible to a respiratory disease or condition, said method comprises simultaneously, concurrently, separately or sequentially administering to the patient an effective amount of a b-adrenergic receptor antagonist and a corticosteroid.