Abstract: A compound, or a pharmaceutically acceptable salt or ester thereof, comprising (i) a CB1 receptor mediating scaffold and (ii) a second therapeutic scaffold.

Abstract: The invention relates to compositions and methods for treating kidney and muscle dysfunction that involves use of therapeutic amounts of N-acetyl mannosamine.

Abstract: The invention relates to compositions and methods for treating kidney and muscle dysfunction that involves use of therapeutic amounts of N-acetyl mannosamine.

Abstract: The invention relates to compositions and methods for treating kidney and muscle dysfunction that involves use of therapeutic amounts of N-acetyl mannosamine.

Abstract: The invention relates to compositions and methods for treating kidney and muscle dysfunction that involves use of therapeutic amounts of N-acetyl mannosamine.

Abstract: A compound, or a pharmaceutically acceptable salt or ester thereof, comprising (i) a CB1 receptor mediating scaffold and (ii) a second therapeutic scaffold.

Abstract: A method is provided for the treatment of vision problems in a subject suffering from one of various forms of albinism, including, for example, oculocutaneous albinism types OCA1a and OCA1b, as well as ocular albinism type 1, resulting from mutations in the GPR143 gene, as well as the OCA2, OCA3 or OCA4 genes, by administering to the subject a pharmaceutical composition comprising a therapeutically effective amount of the compound (2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione), also known as NTBC for a sufficient period of time. The administration of NTBC is believed to increase the amount of pigmentation in the subject and alleviate certain symptoms caused by lack of pigmentation in the eye tissues. Also described are methods of use of NTBC for increasing the pigmentation of a subject for cosmetic purposes, by administering to the subject a therapeutically effective amount of NTBC.

Abstract: Methods are disclosed for diagnosing a hyposialylation disorder. Methods are also disclosed for determining the effectiveness of a therapeutic agent for treatment of a hyposialylation disorder in a subject. These methods include measuring an amount of monosialylated Thomsen-Friedenreich (ST) antigen and measuring an amount of non-sialylated Thomsen-Friedenreich antigen (T) in a biological sample, such as a serum or plasma sample from the subject and determining the ratio of T to ST. A ratio of T to monosialylated ST of about 0.06 or higher diagnoses the hyposialylation disorder or indicates that the therapeutic agent is not effective for the treatment of the hyposialylation disorder. In other embodiments, a ratio of T to ST less than about 0.06 indicates that the therapeutic agent is effective for the treatment of the hyposialylation disorder, or the subject does not have the hyposialylation disorder.

Abstract: Methods are disclosed for diagnosing a hyposialylation disorder. Methods are also disclosed for determining the effectiveness of a therapeutic agent for treatment of a hyposialylation disorder in a subject. These methods include measuring an amount of monosialylated Thomsen-Friedenreich (ST) antigen and measuring an amount of non-sialylated Thomsen-Friedenreich antigen (T) in a biological sample, such as a serum or plasma sample from the subject, and determining the ratio of T to ST.

Abstract: The invention relates to compositions and methods for treating kidney and muscle dysfunction that involves use of therapeutic amounts of N-acetyl mannosamine.

Abstract: The invention relates to compositions and methods for treating kidney and muscle dysfunction that involves use of therapeutic amounts of N-acetyl mannosamine.

Abstract: A method is provided for the treatment of vision problems in a subject suffering from one of various forms of albinism, including, for example, oculocutaneous albinism types OCA1a and OCA1b, as well as ocular albinism type 1, resulting from mutations in the GPR143 gene, as well as the OCA2, OCA3 or OCA4 genes, by administering to the subject a pharmaceutical composition comprising a therapeutically effective amount of the compound (2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione), also known as NTBC for a sufficient period of time. The administration of NTBC is believed to increase the amount of pigmentation in the subject and alleviate certain symptoms caused by lack of pigmentation in the eye tissues. Also described are methods of use of NTBC for increasing the pigmentation of a subject for cosmetic purposes, by administering to the subject a therapeutically effective amount of NTBC.

Abstract: Methods are disclosed for diagnosing a hyposialylation disorder. Methods are also disclosed for determining the effectiveness of a therapeutic agent for treatment of a hyposialylation disorder in a subject. These methods include measuring an amount of monosialylated Thomsen-Friedenreich (ST) antigen and measuring an amount of non-sialylated Thomsen-Friedenreich antigen (T) in a biological sample, such as a serum or plasma sample from the subject and determining the ratio of T to ST. A ratio of T to monosialylated ST of about 0.06 or higher diagnoses the hyposialylation disorder or indicates that the therapeutic agent is not effective for the treatment of the hyposialylation disorder. In other embodiments, a ratio of T to ST less than about 0.06 indicates that the therapeutic agent is effective for the treatment of the hyposialylation disorder, or the subject does not have the hyposialylation disorder.

Abstract: A method is provided for the treatment of vision problems in a subject suffering from one of various forms of albinism, including, for example, oculocutaneous albinism types OCA1a and OCA1b, as well as ocular albinism type 1, resulting from mutations in the GPR143 gene, as well as the OCA2, OCA3 or OCA4 genes, by administering to the subject a pharmaceutical composition comprising a therapeutically effective amount of the compound (2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione), also known as NTBC for a sufficient period of time. The administration of NTBC is believed to increase the amount of pigmentation in the subject and alleviate certain symptoms caused by lack of pigmentation in the eye tissues. Also described are methods of use of NTBC for increasing the pigmentation of a subject for cosmetic purposes, by administering to the subject a therapeutically effective amount of NTBC.

Abstract: Disclosed are a method of treating or preventing a disorder in a mammal comprising administering to the mammal an adenosine receptor agonist or an adenosine receptor antagonist, either alone or in combination, in an amount effective to treat or prevent medial vascular or joint capsule calcification. Disclosed are methods of detecting or diagnosing a vascular or joint capsule calcification disorder, as well as a nucleic acid comprising a mutation in one or more exons of human NT5E selected from the group consisting of Exon 3, Exon 5, and Exon 9.

Abstract: The invention relates to compositions and methods for treating kidney and muscle dysfunction that involves use of therapeutic amounts of N-acetyl mannosamine.

Abstract: A method for treating a disorder characterized by hyposialylation in a subject that includes administering to the subject an effective amount of (i) a composition comprising N-acetylmannosamine, or a derivative thereof, encapsulated within a liposome, or (ii) a composition comprising N-acetylneuraminic acid, or a derivative thereof, encapsulated within a liposome, wherein the liposome comprises 1,2-dioleoyl-3-trimethylammonium-propane and cholesterol.

Abstract: A method is provided for the treatment of vision problems in a subject suffering from one of various forms of albinism, including, for example, oculocutaneous albinism types OCA1a and OCA1b, as well as ocular albinism type 1, resulting from mutations in the GPR143 gene, as well as the OCA2, OCA3 or OCA4 genes, by administering to the subject a pharmaceutical composition comprising a therapeutically effective amount of the compound (242-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione), also known as NTBC for a sufficient period of time. The administration of NTBC is believed to increase the amount of pigmentation in the subject and alleviate certain symptoms caused by lack of pigmentation in the eye tissues. Also described are methods of use of NTBC for increasing the pigmentation of a subject for cosmetic purposes, by administering to the subject a therapeutically effective amount of NTBC.

Abstract: The invention relates to compositions and methods for treating kidney and muscle dysfunction that involves use of therapeutic amounts of N-acetyl mannosamine.