A novel AI-driven drug-repurposing method, DeepDrug, is used to identify a lead combination of previously FDA-approved drugs to treat AD by targeting the upstream genetic markers along the AD pathology. A three-step methodology is used. First, a heterogeneous biomedical graph is constructed comprising complex and interconnected genes, proteins, and drug information to capture the network characteristics of the AD pathology, considering the expert known associations between different AD pathways and utilizing node weighting and edge weighting and direction. Second, the curated graph is taken as an input to an artificial intelligence (AI)-driven graphical neural network (GNN) framework, with embeddings of drug and gene nodes as the outputs. Third, a drug scoring and selection analysis is conduced to generate the drug-gene scores and identify a lead combination of repurposed AD drug candidates for clinical verification.

Described herein are compositions comprising (a) methyl 3 -((methyl sulfonyl)amino)-2-(((4-phenylcyclohexyl)oxy)methyl)piperidine- 1 -carboxylate or a salt thereof; and (b) an agent for reducing metabolism of (a) and uses thereof.

Described herein are compositions comprising (a) methyl 3 -((methyl sulfonyl)amino)-2-(((4-phenylcyclohexyl)oxy)methyl)piperidine- 1 -carboxylate or a salt thereof; and (b) an agent for reducing metabolism of (a) and uses thereof.

Compositions and methods relating to induction of cell death such as in cancer cells are disclosed. Compounds and related methods for synthesis and use thereof, including the use of compounds in therapy for the treatment of cancer and selective induction of apoptosis in cells are disclosed. Compounds in connection with modification of procaspases such as procaspase-3 are disclosed. In various embodiments, the compounds and compositions are capable of activation of procaspase-3.

Compositions and methods relating to induction of cell death such as in cancer cells are disclosed. Compounds and related methods for synthesis and use thereof, including the use of compounds in therapy for the treatment of cancer and selective induction of apoptosis in cells are disclosed. Compounds in connection with modification of procaspases such as procaspase-3 are disclosed. In various embodiments, the compounds and compositions are capable of activation of procaspase-3.

Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-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.

Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-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.

The present invention provides a biodegradable drug delivery composition comprising a mixture of at least two block copolymers taken among triblock and diblock copolymers comprising: (a) a biodegradable triblock copolymer having the formula: A.sub.v-B.sub.w-A.sub.x wherein A is a polyester and B is polyethylene glycol and v and x are from 1 to 3,000 and w is from 3 to 300 and v=x or v≠x; (b) a biodegradable diblock copolymer having the formula: C.sub.y-A.sub.z wherein A is a polyester and C is an end-capped polyethylene glycol and y and z are the number of repeat units with y=2 to 250 and z=1 to 3,000; wherein the weight ratio between (a) and (b) is 1:19 to 5:1; and for at least one of the copolymers according to (a) or (b) A is poly(ε-caprolactone-co-lactide); and (c) at least one pharmaceutically active ingredient.

The present invention provides a biodegradable drug delivery composition comprising a mixture of at least two block copolymers taken among triblock and diblock copolymers comprising: (a) a biodegradable triblock copolymer having the formula: A.sub.v-B.sub.w-A.sub.x wherein A is a polyester and B is polyethylene glycol and v and x are from 1 to 3,000 and w is from 3 to 300 and v=x or v≠x; (b) a biodegradable diblock copolymer having the formula: C.sub.y-A.sub.z wherein A is a polyester and C is an end-capped polyethylene glycol and y and z are the number of repeat units with y=2 to 250 and z=1 to 3,000; wherein the weight ratio between (a) and (b) is 1:19 to 5:1; and for at least one of the copolymers according to (a) or (b) A is poly(ε-caprolactone-co-lactide); and (c) at least one pharmaceutically active ingredient.

The present invention relates to sulfonylureas and sulfonylthioureas comprising a 5-membered nitrogen-containing heteroaryl ring attached to the sulfonyl group, wherein the heteroaryl ring is substituted with a nitrogen-containing group R.sup.1′ wherein R.sup.1 contains from 1 to 7 atoms other than hydrogen or halogen. The present invention further relates to salts, solvates and prodrugs of such compounds, to pharmaceutical compositions comprising such compounds, and to the use of such compounds in the treatment and prevention of medical disorders and diseases, most especially by the inhibition of NLRP.sub.3.