Abstract: Methods for treating MPS III diseases are disclosed. The methods involve administering a therapeutically effective amount of a composition comprising ambroxol to a subject with the disease. Also disclosed are pharmaceuticals suitable for treating MPS III diseases.

Abstract: Provided herein are long-acting, non-aqueous pharmaceutically acceptable compositions of active ingredients for subcutaneous injection.

Abstract: Phytol contained in watermelon sprouts is known to have a cancer cell growth inhibiting effect. However, there is a problem that an amount of phytol to be taken for exhibiting cancer cell growth inhibition is large. A cancer cell growth inhibiting composition comprising at least one of compounds having a structure represented by Formula (1), Formula (2), Formula (6), Formula (7) or Formula (8), or a pharmaceutically acceptable salt thereof as main components has a higher cancer cell growth inhibiting effect than phytol.

Abstract: Devices, systems, and methods for a treating dementia or Alzheimer's disease in a subject in need thereof. In one example, combined auditory and visual stimuli having a frequency of about 20 Hz to about 60 Hz, and more specifically about 40 Hz, are non-invasively delivered to the subject to induce synchronized gamma oscillations in at least one brain region of the subject. In particular, pursuant to various treatment and exposure protocols, combined auditory and visual stimulation (as opposed to auditory or visual stimulation alone) promotes a microglia response in the medial prefrontal cortex (mPFC). More generally, combined auditory and visual stimulation induces an extended microglia clustering response in the auditory cortex, the visual cortex, and the mPFC.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are a recombinant acid ?-glucosidase and pharmaceutical composition comprising a recombinant acid ?-glucosidase, wherein the recombinant acid ?-glucosidase is expressed in Chinese hamster ovary (CHO) cells and comprises an increased content of N-glycan units bearing one or two mannose-6-phosphate residues when compared to a content of N-glycan units bearing one or two mannose-6-phosphate residues of alglucosidase alfa. Also provided herein are methods of producing, purifying, and formulating the recombinant acid ?-glucosidase or pharmaceutical composition for administration to a subject and methods of treating a disease or disorder such as Pompe disease using the recombinant acid ?-glucosidase or pharmaceutical composition.

Abstract: An FXR small molecule agonist and a preparation method therefor and a use thereof, having a structure as shown in formula (I). The compound represented by formula (I) has FXR agonistic activity and is capable of preparing drugs for treatment of FXR-related diseases.

Abstract: Some embodiments disclosed herein include a method for increasing an expression level of a gene. The methods include administering an effective amount of a nitroxide antioxidant to a human subject having a decreased expression level of SIRT2, whereby expression level of the gene is increased.

Abstract: The present invention features a method of reducing tactile dysfunction or anxiety in a subject diagnosed with Autism Spectrum Disorder, Rett Syndrome, or Fragile X syndrome by administering a GABAA agent having reduced blood brain barrier or by expressing a nucleic acid encoding an exogenous alpha or beta subunit of a GABAA receptor in dorsal root ganglion neurons in the subject using a vector.

Abstract: Provided herein are methods of treating or ameliorating hypercholemia or a cholestatic liver disease by administering to an individual in need thereof a therapeutically effective amount of an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTI) or a pharmaceutically acceptable salt thereof. Also provided are methods for treating or ameliorating a liver disease, decreasing the levels of serum bile acids or hepatic bile acids, treating or ameliorating pruritis, reducing liver enzymes, or reducing bilirubin comprising administering to an individual in need thereof a therapeutically effective amount of ASBTI or a pharmaceutically acceptable salt thereof.

Abstract: The disclosure provides compounds with warheads and their use in treating medical diseases or disorders, such as viral infections. Pharmaceutical compositions and methods of making various compounds with warheads are provided. The compounds are contemplated to inhibit proteases, such as the 3C, CL- or 3CL-like protease.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: The disclosure provides ionizable amine lipids and salts thereof (e.g., pharmaceutically acceptable salts thereof) useful for the delivery of biologically active agents, for example delivering biologically active agents to cells to prepare engineered cells. The ionizable amine lipids disclosed herein are useful as ionizable lipids in the formulation of lipid nanoparticle-based compositions.

Abstract: Provided are dosing regimens for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-nave Fabry patients. Certain methods comprise administering to the patient about 123 mg free base equivalent of migalastat for improving left ventricular mass and/or improving podocyte globotriaosylceramide.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided is an effective method for using an orally administrable compound having an excellent Aurora A selective inhibitory effect, and a microtubule-targeting drug in combination. The present invention provides a method for treatment of malignant tumor by a combination of 1-(2,3-dichlorobenzoyl)-4-[5-fluoro-6-(5-methyl-1H-pyrazol-3-ylamino)pyridin-2-yl]methyl-4-piperidinecarboxylic acid (compound I) or a salt thereof and a microtubule-targeting drug, wherein the following administration schedule thereof is performed: one cycle involves 7 days; the microtubule-targeting drug is administered once; and the compound I or the salt thereof is administered for 3 consecutive days with cessation of the drug for remaining 4 days.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: In alternative embodiments, provided are uncharged bis-oxime antidotes that cross the blood-brain barrier (BBB) to catalyze the hydrolysis of organophosphate (OP)-inhibited human acetylcholinesterase (hAChE) in the central nerve system (CNS). In alternative embodiments, provided are pumps, devices, subcutaneous infusion devices, continuous subcutaneous infusion devices, infusion pens, needles, reservoirs, ampoules, a vial, a syringe, a cartridge, a disposable pen or jet injector, a prefilled pen or a syringe or a cartridge, a cartridge or a disposable pen or jet injector, a two chambered or multi-chambered pump, a syringe, a cartridge or a pen or a jet injector, comprising a compound as provided herein.

Abstract: Pharmaceutical compositions comprising azelastine or a pharmaceutically acceptable salt of azelastine and melatonin are disclosed. Methods of using the pharmaceutical compositions for treating patients suffering from insomnia are also disclosed.

Abstract: The invention relates to a crystalline form of [(2R,3S,4R,5S)-1-butyl-2-(hydroxymethyl)-piperidine-3,4,5-triol, processes for the preparation thereof, pharmaceutical compositions containing such a crystalline form, and its use as a medicament, especially as glycolipid biosynthesis inhibitor.

Abstract: The present disclosure relates to methods and systems suitable for use in identifying and providing personalised medicine to a patient. In some aspects, systems and method generate a co-therapy regimen for a patient suffering from a disease or condition. An identification of a co-therapy suitable to treat the disease or condition is received. A desired patient endpoint and a patient position are received, wherein the patient position is defined relative to the desired patient endpoint. A dataset relating to the patient is stored. The dataset comprises one or more patient data based on patient-related measurements. The dataset, the patient position and the desired patient endpoint are processed to generate a regimen for the co-therapy. The regimen is stored in a database.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: The invention is directed to substituted piperidine derivatives. Specifically, the invention is directed to compounds according to Formula IIII: wherein A, B, X, Y, L1, L2, L3, R1, R2, R3, R4, R5, R6, R9, z2, z4, z5, and z6 are as defined herein, and salts thereof.

Abstract: The present technology is directed to compositions comprising d-threo-methylphenidate conjugates and/or unconjugated methylphenidate. The present technology also relates to compositions and oral formulations comprising d-threo-methylphenidate conjugated to nicotinoyl-L-serine, and/or a pharmaceutically acceptable salt thereof, and unconjugated methylphenidate and/or a pharmaceutically acceptable salt thereof. The present technology additionally relates to a pharmaceutical kit containing the composition comprising d-threo-methylphenidate conjugated to nicotinoyl-L-serine, and/or a pharmaceutically acceptable salt thereof, and unconjugated methylphenidate and/or a pharmaceutically acceptable salt thereof.

Abstract: A tri-carboxylic compound of Formula (IA) optionally in combination with a compound of Formula (IIA) behaves as a low-VOC, low odor coalescing agent, while a tri-carboxylic compound of Formula (IA) in combination with a compound of Formula (IIB), or alternatively, a tri-carboxylic compound of Formula (IB) in combination with a compound of Formula (IIA) exhibits dual coalescing and plasticizing properties. A tri-carboxylic compound of Formula (IB) optionally in combination with a compound of Formula (IIB) behaves as low-VOC, low odor plasticizing agent.

Abstract: The present invention provides novel compounds as well as compositions and methods using the same for preventing and/or treating degenerative disorders of the central nervous system and/or lysosomal storage disorders. In particular, the present invention provides methods for preventing and/or treating Parkinson's disease, Alzheimer's disease and/or Gaucher's disease.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided is a substituted piperidine compound having an orexin type 2 receptor agonist activity. A compound represented by the formula (I): wherein each symbol is as described in the DESCRIPTION, or a salt thereof has an orexin type 2 receptor agonist activity, and is useful as a prophylactic or therapeutic agent for narcolepsy.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are in vitro and in vivo methods for determining whether a patient with Fabry disease will respond to treatment with a specific pharmacological chaperone.

Abstract: A therapeutic agent for fibrosis, whose active ingredient is constituted by a glucosylceramide synthase inhibitor or lactosylceramide synthase inhibitor, or both, inhibits the nuclear translocation of phosphorylated Smad, thereby treating and preventing fibrosis.

Abstract: Provided is a substituted piperidine compound having an orexin type 2 receptor agonist activity. A compound represented by the formula (I): wherein each symbol is as described in the DESCRIPTION, or a salt thereof has an orexin type 2 receptor agonist activity, and is useful as a prophylactic or therapeutic agent for narcolepsy.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.