Lysosomal dysfunction in neurological and psychiatric disorders
AssigneesThe Johns Hopkins University, Sumitomo Pharma Co., Ltd.
InventorsAkira Sawa, Koko Ishizuka, Kun Yang, Atsushi Saito, Hana Kida, Masakuni Horiguchi, Yoshio Nakai, Tomonori Kobayashi
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Abstract
Compositions which treat lysosomal deficits in lysosomal storage disorders, neuronal ceroid lipofuscinosis, idiopathic/sporadic brain disorders that include neurological and psychiatric disorders, include agents such as troglitazone, rosuvastatin, Compound A or the combination thereof.
Claims27
A method of treating a subject at risk of, or diagnosed with a disease or disorder associated with lysosomal dysfunction, comprising:
- administering to the subject a therapeutically effective dose of one or more agents, wherein the one or more agents modulate the function or activity of a lysosome, thereby treating a subject at risk of, or diagnosed with a disease or disorder associated with lysosomal dysfunction.
(canceled)
A method of treating a subject suffering from or susceptible a lysosomal storage disorder (LSD) or a neuronal ceroid lipofuscinose (NCL), comprising:
- administering to the subject an effective amount of one or more agents that modulate the function or activity of a lysosome.
A method of treating a subject suffering from or susceptible to a lysosomal storage disorder (LSD) or a neuronal ceroid lipofuscinose (NCL), comprising:
- administering to the subject an effective amount of one or more agents selected from:
- i) troglitazone and/or rosuvastatin;
- ii) one or more activators of peroxisome proliferator-activated receptors (PPARs);
- iii) one or more thiazolidinedione compounds;
- iv) one or more statin compounds;
- and/or v) Compound A.
22-49. (canceled)
A method of treating a subject at risk of, or diagnosed with Batten disease, comprising:
- administering to the subject a therapeutically effective amount of one or more activators of peroxisome proliferator-activated receptors (PPARs), one or more thiazolidinedione compounds, and/or one or more statin compounds.
51-53. (canceled)
A method of treating a subject at risk of, or diagnosed with Batten disease, comprising:
- administering to the subject a therapeutically effective amount of one or more small molecule compounds that can modulate the function or activity of a lysosome.
55-58. (canceled)
Dependencies are inferred from the claim text.