Abstract: Novel aminoglycosides, represented by Formulae Ia and Ib, as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.

Abstract: Novel aminoglycosides, represented by Formulae Ia and Ib, as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.

Abstract: Novel aminoglycosides, represented by Formulae Ia and Ib, as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.

Abstract: Compounds, methods and uses of pseudo-trisaccharide aminoglycosides represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification, in the treatment of Rett syndrome are disclosed.

Abstract: Compounds, methods and uses of pseudo-trisaccharide aminoglycosides represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification, in the treatment of Rett syndrome are disclosed.

Abstract: Novel aminoglycosides, represented by Formulae I, Ia, III and IIIa, as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.

Abstract: Novel aminoglycosides, represented by Formulae Ia and Ib, as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.

Abstract: Compounds, methods and uses of pseudo-trisaccharide aminoglycosides represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification, in the treatment of Rett syndrome are disclosed.

Abstract: The disclosure provides, inter alia, methods and reagents for use in measuring the attachment of ubiquitin and ubiquitin-like proteins to a target protein, particularly an E3 protein.

Abstract: The pharmaceutical compositions described comprise a therapeutically effective amount of a ferric compound and at least one bioavailability enhancer for oral delivery. Some pharmaceutical compositions described herein include a suspension which comprises an admixture in solid form of a therapeutically effective amount of a ferric compound and at least one bioavailability enhancer (e.g. a salt of a medium chain fatty acid) and a lipophilic medium. The pharmaceutical compositions may be enteric-coated. Methods of treating or preventing diseases by administering such compositions to affected subjects are also disclosed. The methods of treatment described herein increase the level of iron in the bloodstream of a subject by administering to the subject an effective amount of an oral composition of a ferric iron compound.

Abstract: The pharmaceutical compositions described herein include a suspension which comprises an admixture in solid form of a therapeutically effective amount of a therapeutic agent (such as CCK-8, octreotide), at least one salt of a medium chain fatty acid, a matrix forming polymer and a hydrophobic(lipophilic) medium. A surfactant may be included in the suspension. The pharmaceutical compositions may be formulated in a capsule or tablet for oral delivery. Methods of treating or preventing diseases by administering such compositions to affected subjects are also disclosed.

Abstract: The application discloses compounds that inhibit ubiquitin ligase activity. The application further discloses antiviral compounds, anticancer compounds, and compounds useful for the treatment of neurological disorders.

Abstract: The disclosure provides, inter alia, methods and reagents for use in measuring the attachment of ubiquitin and ubiquitin-like proteins to a target protein, particularly an E3 protein.

Abstract: The application discloses novel polypeptides and nucleic acids involved in a variety of biological processes, including viral reproduction. Related methods and compositions are also described.

Abstract: The application discloses novel polypeptides and nucleic acids involved in a variety of biological processes, including viral reproduction. Related methods and compositions are also described.

Abstract: The application provides novel complexes of POSH polypeptides and POSH asoociated kinases. The application also provides methods and compositions for treating a POSH-associated diseases such as viral disorders and cancer.

Abstract: The application discloses novel polypeptides and nucleic acids involved in a variety of biological processes, including viral reproduction. Related methods and compositions are also described.

Abstract: The application discloses novel polypeptides and nucleic acids involved in a variety of biological processes, including viral reproduction. Related methods and compositions are also described.

Abstract: The application provides novel complexes of Cbl-b polypeptides and Cbl-b-associated proteins. The application also provides methods and compositions for treating Cbl-b-associated diseases such as viral disorders.

Abstract: The application discloses compounds that inhibit ubiquitin ligase activity. The application further discloses antiviral compounds, anticancer compounds, and compounds useful for the treatment of neurological disorders.