One aspect of the invention provides a method for treatment of a lysosomal storage disorder. The method may include administering to a subject in need of such treatment a composition including a therapeutically effective amount of an agent that mediates upregulation of Transcription Factor EB. In one embodiment, the composition includes a fibrate, such as gemfibrozil or fenofibrate. In another embodiment, the composition also includes all-trans retinoic acid or vitamin A.

A composition for treating, ameliorating, or preventing an infection in a mammalian urinary tract is provided. The composition comprises a monosaccharide comprising a D-mannose, an oligosaccharide comprising an isolated human milk oligosaccharide (HMO), a flavonoid comprising an A-type proanthocyanidin, quercetin, a vitamin comprising ascorbic acid, a green tea extract, and optionally a triterpenoid comprising ursolic acid. A method of treating, ameliorating, or preventing an infection in a urinary tract of a mammal is also provided, and comprises administering the composition to the mammal.

A composition for treating, ameliorating, or preventing an infection in a mammalian urinary tract is provided. The composition comprises a monosaccharide comprising a D-mannose, an oligosaccharide comprising an isolated human milk oligosaccharide (HMO), a flavonoid comprising an A-type proanthocyanidin, quercetin, a vitamin comprising ascorbic acid, a green tea extract, and optionally a triterpenoid comprising ursolic acid. A method of treating, ameliorating, or preventing an infection in a urinary tract of a mammal is also provided, and comprises administering the composition to the mammal.

A method of treating an infection of a lung in a patient in need thereof according to an embodiment includes administering to the patient an effective amount of a compound having the formula:

##STR00001##

or a pharmaceutically acceptable salt thereof.

A method of treating an infection of a lung in a patient in need thereof according to an embodiment includes administering to the patient an effective amount of a compound having the formula:

##STR00001##

or a pharmaceutically acceptable salt thereof.

Pharmaceutical compositions comprising a vehicle, a solubilizer, and a surfactant are disclosed to be used in conjunction with non-chemotherapeutic active pharmaceutical ingredients which are soluble in water but subject to hydrolysis to some extent. Such non-chemotherapeutic active pharmaceutical ingredients may include amitriptyline, a derivative thereof, or a combination thereof. The pharmaceutical compositions may be administered as an oral solution. Other embodiments are directed towards methods of using and methods of making such formulations.

Embodiments of the present invention relate to a method of treating cancer in a patient comprising administering an immune checkpoint inhibitor to the patient, wherein the patient has been diagnosed with an FGFR-genetically altered cancer, and has been pre-treated with an FGFR inhibitor, such as erdafitinib.

Embodiments of the present invention relate to a method of treating cancer in a patient comprising administering an immune checkpoint inhibitor to the patient, wherein the patient has been diagnosed with an FGFR-genetically altered cancer, and has been pre-treated with an FGFR inhibitor, such as erdafitinib.

Provided in this disclosure, in some embodiments, are methods and compositions for treating maple syrup urine disease (MSUD) and other conditions characterized by excessive branched-chain amino acids.

Described herein are compounds, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or disorders associated with a defect in glyoxylate metabolism, for example a disease or disorder associated with the enzyme glycolate oxidase (GO) or alterations in oxalate metabolism. Such diseases or disorders include, for example, disorders of glyoxylate metabolism, including primary hyperoxaluria, that are associated with production of excessive amounts of oxalate.