Abstract: The invention provides methods for modulating the activity of farnesoid X receptors (FXRs) using specific FXR agonists, in particular for treating or preventing liver diseases and disorders.

Abstract: Described herein are compounds of Formula (I), pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof. Compounds of the present invention are useful for inhibiting PRMT5 activity. Methods of using the compounds for treating PRMT5-mediated disorders are also described.

Abstract: The present disclosure relates generally to compositions and methods of treating neoplastic diseases or cancers, such as glioblastoma and non-Hodgkin's lymphomas, or other cancers in which the subject suffers from an advanced solid tumor, comprising a combination of, or administering a combination of, a bromodomain and extra-terminal protein (BET) inhibitor and at least one chemotherapeutic agent, which does not inhibit BET directly. The BET inhibitor/chemotherapeutic agent combination, or combination therapy, can yield synergistic effects, thereby increasing the effectiveness of the cancer treatment as compared with the administration of either the BET inhibitor or the chemotherapeutic agent alone.

Abstract: The present invention relates to new pharmaceutical compositions comprising benzoquinoline compounds, and methods to inhibit vesicular monoamine transporter 2 (VMAT2) activity in a subject for the treatment of chronic hyperkinetic movement disorders.

Abstract: The present invention relates to methods of treating patients with WHIM syndrome or related disorders, such as myelokathexis, in which X4P-001 is administered in order to reduce the activity of CXCR4. The methods demonstrate surprising effectiveness, with comparatively little toxicity.

Abstract: This invention provides a method that combines a checkpoint inhibitor and a glucocorticoid receptor modulator to treat cancer, e.g., a checkpoint inhibitor sensitive cancer.

Abstract: The present invention relates to therapeutic immunoconjugates comprising SN-38 attached to an anti-HLA-DR antibody or antigen-binding antibody fragment. The immunoconjugate may be administered at a dosage of between 3 mg/kg and 18 mg/kg, preferably 4, 6, 8, 9, 10, 12, 16 or 18 mg/kg, more preferably 8, 10 or 12 mg/kg. When administered at specified dosages and schedules, the immunoconjugate can reduce solid tumors in size, reduce or eliminate metastases and is effective to treat cancers resistant to standard therapies, such as radiation therapy, chemotherapy or immunotherapy. The methods and compositions are particularly useful for treating AML, ALL or multiple myeloma.

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: Provided herein, inter alia, are compositions and methods for generating a immune response in an individual and/or inducing the expression of neoantigens on the surface of abnormal (such as proliferative) cells via inhibition of nonsense-mediated decay (NMD) of messenger RNAs (mRNAs) bearing premature termination codons (PTCs).

Abstract: The present invention relates to a comprising (A) an active ingredient which is hardly soluble in water, but soluble in a C1-4 lower alcohol that may contain 30 vol % or less water, (B) polyvinyl alcohol having a saponification rate of 55-99%, and (C) a non-ionic surfactant, wherein the (A) active ingredient has a mean particle size of 10-300 nm, and a process thereof.

Abstract: Embodiments concern methods and small molecule compositions for selectively inhibiting RAD51-mediated D-loop formation while preserving RAD51's ability to form nucleoprotein filaments. The selective RAD51 D-loop formation activity inhibitors DNA repair while minimizing replication-associated toxicity in normal tissue.

Abstract: The present invention is related to a topical Minoxidil foam or spray composition, wherein the excipient 1,3-propanediol used for the first time as a solvent can be substituted with polyethylene glycol and an exemplary production method of Minoxidil foam or spray composition using 1,3-propanediol.

Abstract: Disclosed are methods of alleviating or preventing one or more symptoms associated with fragile X syndrome in an individual in need thereof via administration of a therapeutically effective amount of a GABA(A) alpha 2 and/or 3 partial agonist. The one or more symptoms may include impaired functional communication, anxiety, inattention, hyperactivity, sensory reactivity, autonomic nervous system dysregulation, aberrant eye gaze, self-injury, aggression, seizures, EEG abnormalities, including but not limited to, abnormal spectral analysis, event related potentials which may include auditory and visual responses, abnormalities in cortical responses as evoked by transcranial magnetic stimulation including resting and active motor thresholds and abnormal responses in measures of cortical inhibition and excitation, aberrant impaired cognitive function, compromised daily living skills, or a combination thereof.

Abstract: The invention relates to a product containing the compound of formula (I) below (I) or a pharmaceutically acceptable salt of this compound, in combination with at least one compound having PDE5-inhibitory properties, or a pharmaceutically acceptable salt thereof, for therapeutic use, simultaneously, separately or over a period of time, in the treatment of a disease wherein vasoconstriction is involved.

Abstract: Described herein are methods of preventing or reducing fibrosis comprising administering CSF1R inhibitors, coating formulations comprising CSF1R inhibitors, coatings comprising CSF1R inhibitors for implantable medical devices, CSF1R inhibitor coated implantable medical devices, as well as corresponding embodiments comprising additional agents.

Abstract: The present disclosure is directed to methods and compositions that are useful in combating the spread of Zika virus infections, such as, but not limited to, methods and compositions for treating, preventing or suppressing a Zika virus infection in a subject. The methods comprise administering to the subject an effective amount of a compound of the disclosure, or a composition (such as a pharmaceutical composition) comprising a compound of the disclosure.

Abstract: The present invention relates to a method of increasing blood brain barrier permeability in a subject. This method involves selecting a subject who would benefit from increased blood brain barrier permeability and subjecting the selected subject to a treatment. That treatment increases adenosine level and/or bioavailability, modulates adenosine receptors, and/or increases CD73 level and/or activity under conditions effective to increase blood brain barrier permeability in the subject. Methods of decreasing blood brain barrier permeability in a subject, treatment of a subject for a disorder or condition of the central nervous system, and screening compounds effective in increasing blood brain barrier permeability, as well as pharmaceutical agents are also disclosed.

Abstract: The invention provides methods that relate to a novel therapeutic strategy for the treatment of hematological malignancies and inflammatory diseases. In particular, the method comprises administering a compound of formula A, wherein R is H, halo, or C1-C6 alkyl; R? is C1-C6 alkyl; or a pharmaceutically acceptable salt thereof; and optionally a pharmaceutically acceptable excipient; and administering at least one additional therapeutic agent.

Abstract: The invention provides a series of chlorobenzene substituted azaaryl compounds having activity in inhibiting cancer cell growth and low toxicity to normal cells. Particularly, the compounds of the invention have stronger inhibition effect on bladder cancer and liver cancer.

Abstract: The present invention relates to a pharmaceutical combination which comprises (a) a phosphatidylinositol 3-kinase inhibitor or pharmaceutically acceptable salt thereof, and (b) at least one c-Met receptor tyrosine kinase inhibitor or pharmaceutically acceptable salt thereof, for simultaneous, separate or sequential administration for the treatment of a proliferative disease, particularly a c-Met dependent proliferative disease; a pharmaceutical composition comprising such combination; a method of treating a subject having a proliferative disease comprising administration of said combination to a subject in need thereof; use of such combination for the treatment of proliferative disease; and a commercial package comprising such combination.

Abstract: Methods and kits are provided for administering a hypoxia-activated bioreductive agent by applying the agent locally to liver tissue when hepatic arterial pressure is reduced. Local arterial application when pressure is reduced tissue prolongs the duration of tissue exposure to the hypoxia-activated bioreductive agent. Hepatic pressure may be reduced proximal to the liver tissue. In some embodiments, local application may be followed by embolization when arterial pressure is reduced. Embolization under reduced pressure may decrease complications associated with backflow and increase the efficacy of the hypoxia-activated agent.

Abstract: The present invention generally relates to the transdermal delivery of various compounds. In some aspects, transdermal delivery may be facilitated by the use of a hostile biophysical environment. One set of embodiments provides a composition for topical delivery comprising a phosphodiesterase type 5 inhibitor and/or a salt thereof, and optionally, a hostile biophysical environment and/or a nitric oxide donor. In some cases, the composition may be stabilized using a combination of a stabilization polymer (such as xanthan gum, KELTROL® BT and/or KELTROL® RD), propylene glycol, and a polysorbate surfactant such as Polysorbate 20, which combination unexpectedly provides temperature stability to the composition, e.g., at elevated temperatures such as at least 40° C. (at least about 104° F.), as compared to compositions lacking one or more of these.

Abstract: Disclosed is a method for treating ear disorders, including the administration of (+)-azasetron, or a pharmaceutically acceptable salt and/or solvate thereof.

Abstract: The disclosure provides a method of treating a medulloblastoma in a subject in need thereof comprising administering to the subject a therapeutically-effective amount of an enhancer of a zeste homolog 2 (EZH2) inhibitor. In a preferred embodiment of this method, the subject is pediatric and the EZH2 inhibitor is Tazemetostat.

Abstract: Methods for enhancing memory and/or learning and prevent neurodegeneration by administration of certain heterocyclic and aromatic compounds are described. The methods are particularly useful for treating patients suffering from a neurodegenerative disease such as (without limitation) Alzheimer's, Parkinsons's, Lou Gehrig's (ALS) disease or memory or learning impairment. A neuronal human cell-based assay that assess NF-kB gene up-regulation using a luciferase reporter is also provided that screens for compounds useful in methods for enhancing memory or learning.

Abstract: Provided herein are (alpha-substituted aralkylamino or heteroarylalkylamino) pyrimidinyl and 1,3,5-triazinyl benzimidazoles, e.g., a compound of Formula I, and their pharmaceutical compositions, preparation, and use as agents or drugs for treating proliferative diseases.

Abstract: The present disclosure teaches methods for treating and preventing a variety of neurodegenerative conditions and symptoms associated therewith, including Alzheimer's disease (AD) and idiopathic Parkinson's disease (iPD), by utilizing adrenoceptor antagonists. Adrenoceptor antagonists that can be used include ?-blockers, such as acebutolol, betaxolol, bisopropolol, bopindolol, carvedilol, metoprolol, oxprenolol, propranolol, and timolol. The present disclosure also teaches methods for diagnosing and monitoring the progression of iPD.

Abstract: The present invention relates to methods and pharmaceutical compositions for the treatment of inflammatory bowel diseases.

Abstract: Methods and compositions are provided for the prevention and/or treatment of symptoms associated with lipid accumulation disorders caused by attenuated leptin activity and by lipid storage disorders. The methods involve administering at least one oxygenated cholesterol sulfate (OCS) to a subject with the disorder. The disorder may be acquired or congenital (hereditary).

Abstract: Provided herein are, inter alia, methods and compositions for treating cancer in a subject in need thereof comprising a therapeutically effective amount of a stilbenoid compound and a progestin.

Abstract: The present invention refers to a new enzymatic process for obtaining 17?-monoesters of cortexolone and/or its 9,11-dehydroderivatives starting from the corresponding 17?,21-diesters which comprises an enzymatic alcoholysis reaction. Furthermore, the present invention refers to new crystalline forms of cortexolone-17?-propionate and 9,11-dehydro-cortexolone 17?-butanoate.

Abstract: The disclosure teaches precisely engineered biodegradable drug delivery systems and methods of making and utilizing such systems. In aspects, the biodegradable drug delivery systems taught herein comprise intravitreal ocular implants suitable for delivery of corticosteroids to the posterior segment of a human eye. The intravitreal ocular implants described herein have a desired extended drug release profile suitable for treating inflammation of the human eye.

Abstract: Methods for treating vitamin D insufficiency and secondary hyperparathyroidism in patients having CKD comprising administering repeat doses of 25-hydroxyvitamin D are disclosed. The methods comprise administering 25-hydroxyvitamin D in an amount effective to safely raise the patient's serum 25-hydroxyvitamin D level to greater than 90 ng/ml and/or to control the patient's serum ratio of 25-hydroxyvitamin D to 24,25-dihydroxyvitamin D to less than 20.

Abstract: The invention is directed to compositions of vitamin D having enhanced bioavailability and enhanced stability. Methods of making and using the compositions of the invention are contemplated and disclosed.

Abstract: The present invention is directed toward the use of thyromimetic compounds that are thyroid receptor ligands, pharmaceutically acceptable salts thereof; and to prodrugs of these compounds for preventing, treating, or ameliorating fatty liver diseases such as steatosis, non-alcoholic fatty liver disease, and non-alcoholic steatohepatitis.

Abstract: The instant invention relates to a composition useful as a nutritional supplement comprising acetylated neuraminate (NeuAc), and/or a compound selected from N-acetyl mannosamine (ManNac), at least one or more compounds of the NeuAc biosynthetic pathway, substrates, products, or derivative thereof, or a mixture thereof; and a divalent metal ion. The composition of the invention permits to increase serum and or cellular NeuAc content in a subject.

Abstract: Provided herein are complexes comprising amphotericin B (AmB) or derivatives and sterols. Also provided herein are methods of treating cystic fibrosis using AmB or complexes comprising AmB or derivatives and sterols.

Abstract: Disclosed are compositions and methods for treating, amelioriating, reversing and/or preventing (acting as a prophylaxis): a respiratory condition involving an infection or an inflammation, or any lung condition involving inflammation or infection, e.g., of a respiratory mucosa, and/or an infection or an inflammation of an underlying muscle of the respiratory tract; or, an asthma; a bronchitis; a sinusitis or rhinosinusitis; an infection of a sinus; chronic obstructive airway disease; emphysema; chronic bronchitis; pneumonia; or, a bronchiectasis. In alternative embodiments, the therapeutic combination comprises an orally administered Amphotericin B or equivalent antifungal alone, or a combination of Amphotericin B and: one antibiotic; two antibiotics; three antibiotics; or, four or more antibiotics. In alternative embodiments, these compositions and methods are dosaged and administered to children in need thereof.

Abstract: Mithramycin side chain carboxylic acid (MTM-SA) derivative are provided, which include a substituted amino acid derivative, a substituted amino acid dipeptide derivative, or an unsubstituted dipeptide derivative. The MTM-SA derivatives are useful for treatment of cancer or neuro-diseases associated with an aberrant erythroblast transformation-specific transcription factor. Unique MTM-SA derivatives have increased selectively toward ETS transcription factor.

Abstract: This disclosure relates to certain N4-hydroxycytidine derivatives, compositions, and methods related thereto. In certain embodiments, the disclosure relates to the treatment or prophylaxis of a Zika virus infection.

Abstract: The disclosure relates to combinations comprising inhibitors of human histone methyltransferase DOT1L and one or more therapeutic agents, particularly anticancer agents, and methods of combination therapy for administering to subjects in need thereof for the treatment of cancer.

Abstract: The present disclosure provides, in some aspects, nucleic acid-based biosynthetic modules for the production of ribonucleic acid (RNA) and other biopolymers.

Abstract: The present invention relates generally to methods and compositions for gene therapy and immunotherapy that activate gamma delta T-cells, and in particular, can be used in the treatment of various cancers and infectious diseases.

Abstract: The present invention generally relates to treatment of iron-related conditions with iron carbohydrate complexes. One aspect of the invention is a method of treatment of iron-related conditions with a single unit dosage of at least about 0.6 grams of elemental iron via an iron carbohydrate complex. The method generally employs iron carbohydrate complexes with nearly neutral pH, physiological osmolarity, and stable and non-immunogenic carbohydrate components so as to rapidly administer high single unit doses of iron intravenously to patients in need thereof.

Abstract: The present disclosure provides a composition comprising Compound 1, or a pharmaceutically acceptable salt thereof, cyclodextrin, and, optionally, pH adjusting agents.

Abstract: The invention generally relates to methods of treating short telomere disorders using known compounds and pharmaceutical compositions comprising same. More specifically, the disclosed methods comprise, in one aspect, the step of administering to the subject an effective amount of at least one lithium compound or a pharmaceutically acceptable salt thereof, thereby treating the subject for the short telomere disorder. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

Abstract: Provided herein are formulations for topical and/or transdermal administration, and methods of using these formulations for the treatment of proliferative diseases related to cancer such as cancers and related conditions, and solid minors. Also provided are formulations for topical and/or transdermal administration, and methods of using these formulations for melasma, gout, skin disorders, and other diseases and disorders described herein as well as methods for modulating the pH (e.g. raising) of a tissue or microenvironment proximal to a tumor, modulating pH, or improving the effectiveness of know chemotherapeutic agents, immunotherapy and the like for the prevention, treatment of cancers and related conditions described herein.

Abstract: Compositions comprising from about 40 weight parts to about 1000 weight parts of a botanical nitrate source; from about 20 weight parts to about 500 weight parts of a botanical source of nitrite reduction activity; and from about 4 weight parts to about 100 weight parts of a nitrite salt. Use of said composition in methods of reducing triglycerides or reducing C-reactive protein levels are also provided.

Abstract: An ophthalmic composition or dosage form can include a therapeutically effective amount of a cross-linking agent and a pharmaceutically acceptable carrier. The composition or dosage form can be used to treat an ophthalmic condition by administering a therapeutically effective amount of the composition to an eye of a subject during a treatment period.

Abstract: Methods and compositions are provided for combined transplantation of a solid organ and hematopoietic cells to a recipient, where tolerance to the graft is established through development of a persistent mixed chimerism. An individual with persistent mixed chimerism, usually for a period of at least six months, is able to withdraw from the use of immunosuppressive drugs after a period of time sufficient to establish tolerance.