A61K31/7008

MULTIPLE FOLATE FORMULATION AND USE THEREOF
20170274002 · 2017-09-28 ·

A multiple folate composition comprising the following three different forms of folate: a folic acid (a salt or ester thereof); a folinic acid (a salt or ester thereof); and a 5-methyl-tetrahydrofolicacid (a salt or ester thereof) and other non-folate ingredients. The composition is useful as a nutritional supplement or medication in the treatment of a folate deficiency and sequella thereof and/or in conditions responsive to administration of a metabolically useful folate. The compositions are particularly of use in patients who have impaired or reduced ability to convert folic acid to its metabolically active forms and in the treatment of depression, particularly in pregnant women or women who may become pregnant.

Methods Of Treatment

Disclosed herein are combinations of an OX40 modulator and a TLR4 modulator, pharmaceutical compositions thereof, uses thereof, and methods of treatment comprising administering said combination, including uses in cancer.

Methods Of Treatment

Disclosed herein are combinations of an OX40 modulator and a TLR4 modulator, pharmaceutical compositions thereof, uses thereof, and methods of treatment comprising administering said combination, including uses in cancer.

COMPOSITIONS AND METHODS FOR TREATING MICROBIAL INFECTIONS

Embodiments of the presently-disclosed subject matter include activators of HssRS that induce endogenous heme biosynthesis by perturbing central metabolism. These molecules are toxic to fermenting S. aureus, including clinically relevant small colony variants (SCVs). The utility of targeting fermenting bacteria is exemplified by the fact that this compound prevents the emergence of antibiotic resistance, enhances phagocyte killing, and reduces S. aureus pathogenesis. This small molecule is a powerful tool not only for studying bacterial heme biosynthesis and central metabolism, but also establishes targeting of fermentation as a viable antibacterial strategy.

COMPOSITIONS AND METHODS FOR TREATING MICROBIAL INFECTIONS

Embodiments of the presently-disclosed subject matter include activators of HssRS that induce endogenous heme biosynthesis by perturbing central metabolism. These molecules are toxic to fermenting S. aureus, including clinically relevant small colony variants (SCVs). The utility of targeting fermenting bacteria is exemplified by the fact that this compound prevents the emergence of antibiotic resistance, enhances phagocyte killing, and reduces S. aureus pathogenesis. This small molecule is a powerful tool not only for studying bacterial heme biosynthesis and central metabolism, but also establishes targeting of fermentation as a viable antibacterial strategy.

DEVICE AND METHOD TO TREAT OR PREVENT JOINT DEGENERATION

A method for the delivery of aggrecan precursors to the joint of a subject, comprising the steps of: a) applying a composition comprising one or more aggrecan precursors chosen from the list comprising hyaluronic acid, glucosamine sulfate and/or chondroitin sulfate to the skin of the subject on or near a joint, wherein the composition is applied between the skin and a flexible magnetic film.

DEVICE AND METHOD TO TREAT OR PREVENT JOINT DEGENERATION

A method for the delivery of aggrecan precursors to the joint of a subject, comprising the steps of: a) applying a composition comprising one or more aggrecan precursors chosen from the list comprising hyaluronic acid, glucosamine sulfate and/or chondroitin sulfate to the skin of the subject on or near a joint, wherein the composition is applied between the skin and a flexible magnetic film.

Dosing Regimens for the Treatment of Lysosomal Storage Diseases Using Pharmacological Chaperones
20220031681 · 2022-02-03 · ·

The present invention provides dosing regimens for administering pharmacological chaperones to a subject in need thereof. The dosing regimens can be used to treat disorders caused by improper protein misfolding, such as lysosomal storage disorders.

Targeting Ligands

Described are novel targeting ligands that may be linked to compounds, such therapeutic compounds that are useful in directing the compounds to the in vivo target. The targeting ligands disclosed herein can serve to target expression-inhibiting oligomeric compounds, such as RNAi agents, to liver cells to modulate gene expression. The targeting ligands disclosed herein, when conjugated to a therapeutic compound, may be used in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Compositions including the targeting ligands disclosed herein when linked to expression-inhibiting oligomeric compounds are capable of mediating expression of target nucleic acid sequences in liver cells, such as hepatocytes, which may be useful in the treatment of diseases or conditions that respond to inhibition of gene expression or activity in a cell, tissue, or organism.

Targeting Ligands

Described are novel targeting ligands that may be linked to compounds, such therapeutic compounds that are useful in directing the compounds to the in vivo target. The targeting ligands disclosed herein can serve to target expression-inhibiting oligomeric compounds, such as RNAi agents, to liver cells to modulate gene expression. The targeting ligands disclosed herein, when conjugated to a therapeutic compound, may be used in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Compositions including the targeting ligands disclosed herein when linked to expression-inhibiting oligomeric compounds are capable of mediating expression of target nucleic acid sequences in liver cells, such as hepatocytes, which may be useful in the treatment of diseases or conditions that respond to inhibition of gene expression or activity in a cell, tissue, or organism.