Abstract: Certain embodiments provide a method of treating Hunter syndrome in a subject in need thereof, comprising administering to the subject a therapeutically effective dose of a pharmaceutical composition comprising an ETV:IDS protein.

Abstract: Provided herein are proteins, which are capable of being transported across the blood-brain barrier (BBB) and comprise sulfoglucosamine sulfohydrolase (SGSH) enzyme-Fc fusion polypeptides. Certain embodiments also provide methods of using such proteins to treat Sanfilippo syndrome A.

Abstract: The present disclosure relates generally to LRRK2 inhibitors, or a pharmaceutically acceptable salt, deuterated analog, prodrug, tautomer, stereoisomer, or mixture of stereoisomers thereof, and methods of making and using thereof.

Abstract: The present disclosure relates generally to compounds and compositions, and their use as kinase inhibitors.

Abstract: The present disclosure relates to modified release formulations of 2-methyl-2-(3-methyl-4-(4-(methylamino)-5-(trifluoromethyl)pyrimidin-2-ylamino)-IH-pyrazol-1-propanenitrile or solvates, tautomers, and pharmaceutically acceptable salts thereof, and methods of treatment with the modified release formulations.

Abstract: Provided herein are progranulin variants and fusion proteins that comprise a progranulin variant and an Fc polypeptide. Methods of using such proteins to treat progranulin-associated disorders (e.g., a neurodegenerative disease, such as frontotemporal dementia (FTD)) are also provided herein.

Abstract: The present disclosure relates generally to LRRK2 inhibitors, or a pharmaceutically acceptable salt, isotopically enriched analog, tautomer, stereoisomer, mixture of stereoisomers, prodrug, or pharmaceutical composition thereof, and methods of making and using thereof.

Abstract: Provided herein are progranulin variants and fusion proteins that comprise a progranulin variant and an Fc polypeptide. Methods of using such proteins to treat progranulin-associated disorders (e.g., a neurodegenerative disease, such as frontotemporal dementia (FTD)) are also provided herein.

Abstract: In one aspect, antibodies that specifically bind to a human Tau protein are provided. In some embodiments, an anti-Tau antibody recognizes an epitope within residues 111-125 of full-length human Tau, an epitope within residues 251-270 and/or residues 346-360 of full-length human Tau, or an epitope within residues 186-205 of full-length human Tau. In some embodiments, an anti-Tau antibody specifically binds to phosphorylated human Tau, unphosphorylated human Tau, and/or multiple splice isoforms of human Tau. An anti-Tau antibody disclosed herein may also include one or two modified Fc polypeptides.

Abstract: Certain embodiments provide a pharmaceutical composition comprising: a protein molecule comprising an ERT enzyme-Fc fusion polypeptide and a modified Fc polypeptide; a buffer; an isotonicity agent; a surfactant; and a stabilizer; wherein the pH of the pharmaceutical composition is about 5.5 to 7.0, as well as methods of use thereof.

Abstract: The present disclosure relates generally to LRRK2 inhibitors, or a pharmaceutically acceptable salt, deuterated analog, prodrug, tautomer, stereoisomer, or mixture of stereoisomers thereof, and methods of making and using thereof.

Abstract: The present disclosure relates generally to LRRK2 inhibitors, or a pharmaceutically acceptable salt, deuterated analog, prodrug, tautomer, stereoisomer, or mixture of stereoisomers thereof, and methods of making and using thereof.

Abstract: In one aspect, antibodies that specifically bind to a human triggering receptor expressed on myeloid cells 2 (TREM2) protein are provided. In some embodiments, the antibody decreases levels of soluble TREM2 (sTREM2). In some embodiments, the antibody enhances TREM2 activity.

Abstract: Compounds having activity as LRRK2 inhibitors are disclosed. The compounds are of formula (I) including stereoisomers, tautomers, pharmaceutically acceptable salts and prodrugs thereof. Methods associated with preparation and use of such compounds, as well as pharmaceutical compositions comprising such compounds, are also disclosed.

Abstract: The present disclosure relates generally to eukaryotic initiation factor 2B modulators, or a pharmaceutically acceptable salt, stereoisomer, mixture of stereoisomers, or prodrug thereof, and methods of making and using thereof.

Abstract: In one aspect, antibodies that specifically bind to a human triggering receptor expressed on myeloid cells 2 (TREM2) protein are provided. In some embodiments, the antibody decreases levels of soluble TREM2 (sTREM2). In some embodiments, the antibody enhances TREM2 activity.

Abstract: The present disclosure relates generally to compounds and compositions, intermediates, processes for their preparation, and their use as kinase inhibitors.

Abstract: The present disclosure relates generally to eukaryotic initiation factor 2B modulators, or a pharmaceutically acceptable salt, stereoisomer, mixture of stereoisomers or prodrug thereof, and methods of making and using thereof.

Abstract: The present disclosure relates generally to methods and compositions for preventing or arresting cell death and/or inflammation.

Abstract: Certain embodiments described herein provide a method for treating dysregulated lipid metabolism and/or inflammation in a mammal in need thereof, comprising administering to the mammal an effective amount of an agonist anti-triggering receptor expressed on myeloid cells 2 (TREM2) antibody.