Abstract: The hemitartrate salt of a compound represented by the following structural formula: (Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.

Abstract: This disclosure relates to methods for improving neural function in a mammal with a proteinopathy comprising administering a therapeutically effective amount of an agent that increases glucocerebrosidase activity in the mammal. Also disclosed are methods for reducing toxic lipids, reducing ?-synuclein, and/or inhibiting the accumulation of protein aggregates in a mammal with a proteinopathy comprising administering a therapeutically effective amount of an agent that increases glucocerebrosidase activity.

Abstract: Provided herein are peptide-linked morpholino (PPMO) antisense oligonucleotides that target the poly CUG repeat tract in the 3? untranslated region of the gene encoding dystrophia myotonica-protein kinase (DMPK) and methods for systemic administration of the same for the treatment of mytonic dystrophy type I (DM1).

Abstract: This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.

Abstract: An article of cookware has a copper core and is surrounded by relatively thinner outer aluminum layers. The outer aluminum layers are preferably anodized to provide a relatively inert hard and scratch resistant durable finish. This anodized finish also readily accepts non-stick finishes.

Abstract: An article of cookware has a copper core and is surrounded by relatively thinner outer aluminum layers. The outer aluminum layers are preferably anodized to provide a relatively inert hard and scratch resistant durable finish. This anodized finish also readily accepts non-stick finishes.

Abstract: The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.

Abstract: The present invention provides a bispecific biologic comprising a ligand specific for CTLA-4 and a ligand specific for a pMHC complex.

Abstract: The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.

Abstract: This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.

Abstract: An antenna structure includes a base, at least one first extending member and at least one second extending member. The base is detachably and pivotally connected to the first extending member, and includes a first transmitting unit. The first extending member is detachably and pivotally connected to the second extending member, and includes a second transmitting unit. The second extending member includes a third transmitting unit. The first transmitting unit is electrically connected to the second transmitting unit, and the second transmitting unit is electrically connected to the third transmitting unit.

Abstract: The hemitartrate salt of a compound represented by the following structural formula: (Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.

Abstract: A portable data storage device (10) includes a universal serial bus (USB) coupling device (1) and an interface device (2) is coupled to the USB coupling device (1). The portable data storage device (10) also includes a memory control device (3) and a non-volatile solid-state memory device (4). The memory control device (3) is coupled between the interface device (2) and the memory device (4) to control the flow of data from the memory device (4) to the USB coupling device (1).

Abstract: An article of cookware has a copper core and is surrounded by relatively thinner outer aluminum layers. The outer aluminum layers are preferably anodized to provide a relatively inert hard and scratch resistant durable finish. This anodized finish also readily accepts non-stick finishes.

Abstract: A method and system for tamper proofing a system of interconnected electronic devices. The method comprises splitting embedded software of each electronic device into at least two executable parts, a stationary part residing in memory of said each electronic device and a non-stationary part residing in memory of another electronic device.

Abstract: This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.

Abstract: This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS).

Abstract: This invention provides various combinations of enzyme replacement therapy, gene therapy, and small molecule therapy for the treatment of lysosomal storage diseases.

Abstract: The present invention relates to methods for treatment of systemic disorders using the lung as a depot organ for transgene delivery. Transfection of the pulmonary epithelium, particularly the deep alveolar cells, or pulmonary endothelial cells, is achieved via local administration of a transgene delivery vector to the lung. The transfected cells express the transgene, and the protein thereby expressed is communicated into the circulatory system. Once entering into the circulatory system, the protein is able to achieve a systemic therapeutic effect.