Abstract: This invention relates to pharmaceutical compositions comprising 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole (C1100), to processes for preparing the compositions, and to various therapeutic uses of the combinations. Also provided is a method of treatment of Duchenne muscular dystrophy or Becker muscular dystrophy using the compositions.

Abstract: This invention relates to pharmaceutical compositions comprising 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole (C1100), to processes for preparing the compositions, and to various therapeutic uses of the combinations. Also provided is a method of treatment of Duchenne muscular dystrophy or Becker muscular dystrophy using the compositions.

Abstract: Disclosed are compounds of formula (I): or a pharmaceutically acceptable salt, derivative, solvate, isomer, tautomer, N-oxide, ester, prodrug, isotope or protected form thereof, which are of use as OGA inhibitors, for example in the treatment of various conditions and diseases, including neurodegenerative disorders.

Abstract: Described are various compounds, in particular iminosugars, and methods for the treatment of proteostatic diseases, in particular lysosomal storage disorders. The compound may be a pharmacoperone of an enzyme selected from: (a) Acid alpha-glucosidase; (b) Acid beta-glucosidase; (c) glucocerebrosidase; (d) alpha-Galactosidase A; (e) Acid beta-galactosidase; (f) beta-Hexosaminidase A; (g) beta-Hexosaminidase B; (h) Acid sphingomyelinase; (i) Galactocerebrosidase; (j) Acid ceramidase; (k) Arylsulfatase A; (l) alpha-L-Iduronidase; (m) Iduronate-2-sulfatase; (n) Heparan N-sulfatase; (o) alpha-N-Acetylglucosaminidase; (p) Acetyl-CoA: alpha-glucosaminide N-acetyltransferase; (q) N-Acetylglucosamine-6-sulfate sulfatase; (r) N-Acetylgalactosamine-6-sulfate sulfatase; (s) Acid beta-galactosidase; (t) Arylsulfatase B; (u) beta-Glucuronidase; (v) Acid alpha-mannosidase; (w) Acid beta-mannosidase; (x) Acid alpha-L-fucosidase; (y) Sialidase; and (z) alpha-N-acetylgalactosaminidase.

Abstract: Described are various compounds and methods for the treatment of flaviviral infections. In particular, alkaloids and imino sugars in arabinose and/or lyxose stereochemical configuration with antiflaviviral activity are described.

Abstract: Described are various compounds and methods for the treatment of disorders arising from aberrant protein folding, including in particular lysosomal storage diseases. In particular, polyhydroxylated alkaloids and imino sugars which are pharmacoperones of an enzyme and which do not bind to a catalytic site of said enzyme are described.

Abstract: A method of detecting an agent that modulates the activity of CCRL2, the method comprising: (a) contacting a CCRL2 polypeptide with a macrophage inflammatory protein-4 (MIP-4) polypeptide in the presence of a candidate agent under conditions, which in the absence of the test agent, permit the binding of the MIP-4 polypeptide to the CCRL2 polypeptide; and (b) determining whether the candidate agent is capable of modulating the interaction between said CCRL2 polypeptide and said MIP-4 polypeptide.

Abstract: Mouse and human utrophin gene promoters are provided. The promoters or fragments and derivatives may be used to control transcription of heterologous sequences, including coding sequences of reporter genes. Expression systems such as host cells containing nucleic acid constructs which comprise a promoter as provided operably linked to a heterologous sequence may be used to screen substances for ability to modulate activity of the utrophin promoter. Substances with such ability may be manufactured and/or used in the preparation of compositions such as medicaments. Up-regulation of utrophin expression may compensate for dystrophin loss in muscular dystrophy patients.

Abstract: Mouse and human utrophin gene promoters are provided. The promoters or fragments and derivatives may be used to control transcription of heterologous sequences, including coding sequences of reporter genes. Expression systems such as host cells containing nucleic acid constructs which comprise a promoter as provided operably linked to a heterologous sequence may be used to screen substances for ability to modulate activity of the utrophin promoter. Substances with such ability may be manufactured and/or used in the preparation of compositions such as medicaments. Up-regulation of utrophin expression may compensate for dystrophin loss in muscular dystrophy patients.