Abstract: Disclosed are small molecules against cereblon to enhance effector T cell function. Methods of making these molecules and methods of using them to treat various disease states are also disclosed.

Abstract: The disclosure provides compositions comprising amino acids, individually and in combination, and methods of making the compositions and methods of using the compositions as pharmaceutically active agents to, inter alia, treat disease in animals, including humans.

Abstract: The present invention provides methods for detecting and/or treating a subject having an aneurysm or at risk for developing an aneurysm. It has been discovered that a key metabolite of the kynurenine (Kyn) pathway, a major route for the metabolism of essential amino acid tryptophan (Trp) into nicotinamide adenine dinucleotide (NAD+), plays a critical role in the formation of aneurysms, for example abdominal aortic aneurysms. In particular, it has been discovered that 3-Hydroxyanthranilic acid (3-HAA), a product of kynureninase (KYNU), plays a causative role in the formation of aneurysms by, for example exerting pro-inflammatory effects on vascular smooth muscle cells. It has further been discovered that elevated levels of 3-HAA are indicative of the presence and/or progression of an aneurysm, and 3-HAA levels correlate with the size (aortic diameter) of the aneurysm.

Abstract: Compositions and methods for the treatment of hair growth and the prevention of hair loss.

Abstract: Provided are a long-acting prodrug of Rasagiline, which has application in the treatment of Central Nervous System diseases such as Parkinson's disease, preparation method and use thereof. The long-acting prodrug of Rasagiline has a structure of formula (I), wherein T is absent, or T is selected from each of R1 and R2 is independently selected from H, D, and alkyl; W is absent, or W is selected from (CH2)n, wherein n is an integer selected from 1 to 15; X is absent, or X is selected from (CH2)m, wherein m is an integer selected from 1 to 10; Y is absent, or Y is selected from —C(?O)NH—, —NHC(?O)—; R3 is selected from substituted or unsubstituted C1-C30 alkyl, substituted or unsubstituted C2-C30 alkenyl, substituted or unsubstituted C2-C30 alkynyl, substituted or unsubstituted C3-C30 cycloalkyl, cholane aliphatic group, —R3a—C(?O)O—R3b, —R3a—OC(?O)—R3b, —R3a—C(?O)NH—R3b, —R3a—NHC(?O)—R3b, —R3a—S(?O)1-2O—R3b and —R3a—OS(?O)1-2—R3b.

Abstract: The present invention provides a method of treating or ameliorating a neurodegenerative disease in a mammal, the method comprising administering to the mammal a therapeutically effective amount of a neurodegenerative disease drug, wherein the drug is a substrate of an ABC transporter inhibitor, wherein the mammal is further administered a therapeutically effective amount of an ABC transporter inhibitor, whereby the neurodegenerative disease is treated in the mammal. In certain embodiments, the neurodegenerative disease comprises at least one selected from the group consisting of spinal cord injury, Alzheimer's disease, Parkinson's disease, Huntington's disease, prion disease, amyotrophic lateral sclerosis, a tauopathy, and chronic traumatic encephalopathy.

Abstract: The present invention concerns a tryptophan metabolite chosen from kynurenic acid, anthranilic acid, quinolinic acid, picolinic acid, quinaldic acid and the mixtures of same or an enantiomer, diastereoisomer, hydrate, solvate, tautomer, racemic mixture or pharmaceutically acceptable salt of same, for use as a drug intended for increasing and/or maintaining muscle mass and/or strength in a mammal, and in particular intended for treating and/or preventing muscle atrophy in mammals and/or for promoting muscle growth in mammals doing exercise and aiming to increase muscle mass and/or quality and/or strength, for preventing the appearance of symptoms of sarcopenia or for rehabilitation following muscle loss and/or for improving recovery time after an intense physical effort.

Abstract: A method of enhancing positive therapeutic effects of a psychedelic, by inducing a positive psychological state in an individual with an empathogen/entactogen of MDMA administered in a dose of 20-200 mg, administering a psychedelic to the individual, and enhancing a positive response to the psychedelic. A composition including an entactogen/empathogen and a psychedelic in the same dosage form.

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

Abstract: Disclosed are methods for treating one or more mammalian cancers, particularly, human breast cancers, including triple-negative breast cancer (TNBC), that employ therapeutically-effective amounts of one or more iNOS pathway-inhibitory compounds, such as L-NMMA, in combination with one or more selected calcium channel antagonists, one or more chemotherapeutic agents, one or more anti-PD-1 antibodies, and one or more doses of ionizing radiation. Also disclosed are pharmaceutical formulations that comprise these compositions, as well as methods for their use in treating refractory, metastatic, and/or relapsed cancers, or, for use in the management or reversal of treatment resistance in one or more types of human breast cancer.

Abstract: A compound, composition, or method for treating cancer is disclosed. The compound (that can be used in the composition and/or method) can include or pharmaceutically acceptable salts or solvates thereof. R1a-R1d, R2-R3 (and R4 where applicable), and A---B can be as defined herein.

Abstract: Potassium chloride cotransporter-2 (KCC2) plays a critical role in brain function, and deficiency in KCC2 has been linked to neurological diseases, psychiatric disorders, and central nervous system injuries. In particular, Rett syndrome (RTT), a severe neurodevelopmental disorder caused by mutations in the X-linked gene Methyl CpG binding Protein 2 (MECP2), has been linked to deficits in KCC2. The disclosure reports the use of CRISPR/Cas9 genome-editing technology to generate stem cell-derived, genetically defined KCC2 reporter human neurons for large-scale compound screening. This screening platform has been utilized to identify a number of small molecule compounds that are capable of enhancing KCC2 expression in both wild-type and RTT neurons, as well as organotypical brain slices cultured from wild-type mice.

Abstract: Provided herein are pharmaceutical compositions comprising deuterated derivatives of psilocybin. The provided compositions may be useful for treating and/or preventing various diseases and conditions, such as mood or psychiatric disorders.

Abstract: The present invention provides a compound capable of being used as an active ingredient of an anti-cancer agent. To provide an anti-cancer agent with few side effects, an object of the present invention is to provide a compound capable of selectively inhibiting the target, i.e., DOCK1, or a salt thereof. The pyridinone compound of the present invention is represented by Formula (1): wherein R1 and R2 are the same or different, and each represents C1-6 alkyl; and R3 is a group represented by, for example, Formula (3) below wherein R5 in the group represented by Formula (3) is hydrogen.

Abstract: The present invention relates to methods and compositions for modulating the blood CSF barrier and for diagnosing, preventing and/or treating leptomeningeal metastasis. In particular embodiments of the invention, the permeability of the blood CSF barrier is modulated by agonists or antagonists of Complement Component 3 (C3) or its receptor.

Abstract: The invention concerns a medicine and a prophylactic medicine for COVID-19 disease. The inventive medicine targets the endosomic, non-endosomic and/or intracellular viral pathways and inhibits them. The best mode of the invention is considered to be the medicine that blocks all three viral pathways. In the best mode the individual dose of a constituent component of the medicine is arranged to a dosage size sufficient to inhibit its designated SARS-CoV-2 viral pathway. This allows the dose of a particular pharmacological agent to be smaller than in a drug with just one kind of pharmacological agent. The best mode of the invention shuts the two cell membrane viral pathways and the one intracellular viral pathway with the minimum efficient dose, thereby preventing drug overdose, and enabling prophylactic or preventive use.

Abstract: Provided herein is a method of administering levoketoconazole, or a pharmaceutically acceptable salt thereof, to a subject in need thereof, wherein the subject is also being administered a multidrug and toxin extrusion transporter 1 (MATE1) substrate or an organic cation transporter 2 (OCT2) substrate.

Abstract: The present invention provides phenylalkylcarboxylic acid compounds and compositions containing such compounds which facilitate the delivery of biologically active agents.

Abstract: Methods of increasing the plasma concentration of resveratrol in a mammal in need thereof by administering to a mammal in need thereof an effective amount of trans-resveratrol and arginine for a period of at least two weeks.

Abstract: Provided are methods and compositions for treating myeloproliferative neoplasms patients carrying an IDH2 mutation and a JAK2 mutation.