Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy. Also provided are kits for detecting the amount of SMN protein in a sample of cerebrospinal fluid.

Abstract: This disclosure relates to a device to mechanically and electrically connect with a touch screen computing device, such as a tablet computer. The device can include a platform that can be moved into and out of physical contact with a surface of a touch screen. During engagement with the surface, the moveable platform electrically interacts with the touch screen (e.g., via capacitive coupling) to enable detection by the touch screen of contact members (e.g., pegs) even in the absence of user contact with the pegs.

Abstract: Formulations and dosage regimens of anti-Blood Dendritic Cell Antigen 2 (BDCA2) antibodies are provided. These formulations and dosage regimens find use in the treatment of BDCA2-associated disorders such as systematic lupus erythematosus, cutaneous lupus eiythernatosus, and discoid lupus erythematosus, and cytokine release syndrome.

Abstract: Compounds of formula (I) or (II) can modulate the activity of S1P receptors.

Abstract: An electrical connector for establishing an electrical connection between two flatly overlapping current conducting elements,

Abstract: Provided herein are deuterium substituted fumarate derivatives and pharmaceutical compositions comprising deuterium substituted fumarate derivatives. Also provided is a method of treating, prophylaxis, or amelioration of a disease, comprising administering to a subject in need of treatment for the disease an effective amount of a deuterium substituted fumarate derivative or a pharmaceutical composition comprising a deuterium substituted fumarate derivative. In one embodiment, the method is a neurodegenerative disease, such as multiple sclerosis, amyotrophic lateral sclerosis, Parkinson's disease, Huntington's disease, or Alzheimer's disease.

Abstract: Provided herein is a method of treating or preventing a disease or disorder (e.g., MS) in a subject in need thereof, comprising (a) administering to the subject a first dose of a pharmaceutical composition comprising a fumarate agent (e.g., DMF) for a first dosing period; (b) administering a vaccine; and (c) administering to the subject a second dose of the pharmaceutical composition for a second dosing period.

Abstract: A limiter for a dispensing device is provided. The limiter includes a body (20) arranged to mount with at least a portion of a dispensing device (300), and a stop extending from the body. The stop (40) is configured to permit movement a plunger of the dispensing device a predetermined distance in a dispensing direction correlated to a desired volume of medium to be delivered from the dispensing device. The stop is configured to prevent further movement of the plunger in the dispensing direction, thereby limiting the amount of medium delivered from the dispensing device. The dispensing device may be a syringe.

Abstract: Disclosed herein are antisense compounds and methods for decreasing SOD-1 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate SOD-1 associated diseases, disorders, and conditions. Such SOD-1 associated diseases include amyotrophic sclerosis (ALS).

Abstract: Provided herein is a method of treating or preventing a disease or disorder (e.g., MS) in a subject in need thereof; comprising (a) administering to the subject a first dose of a pharmaceutical composition comprising a fumarate agent (e.g., DIVEF) for a first dosing period; (b) administering a vaccine; and (c) administering to the subject a second dose of the pharmaceutical composition for a second dosing period.

Abstract: The present invention provides antibodies and antigen-binding molecules thereof which specifically bind the ? and/or ? subunits of the non-active form of the GPIIb/IIIIa receptor. The antibodies and antigen-binding molecules can be genetically fused and/or conjugated to heterologous moieties and used, for example, as targeting moieties. The invention also includes methods for screening for these antibodies, as well as methods of making and methods of using chimeric molecules derived from the antibodies.

Abstract: Provided herein are conjugates comprising targeting moieties such as sugars, folates and cell-penetrating peptides, which can be used for the improved delivery of agents (e.g., nucleic acids, such as oligonucleotides or mRNAs, or other agents) to cells. The invention provides conjugates and compounds comprising targeting moieties, methods for preparing the same, and intermediates useful in their preparation. In another aspect, the present invention provides formulations (e.g., pharmaceutical compositions) comprising the targeting moiety-containing conjugates and compounds. The present invention also provides methods for delivering agents (e.g., nucleic acids such as oligonucleotides or mRNAs) to a cell, methods for treating and/or preventing a disease or condition in a subject, and methods for modulating gene expression in a cell or a subject. Further, provided herein are kits comprising the conjugates, or formulations thereof; and kits for the preparation of conjugates described herein.

Abstract: A metering device that improves the aseptic transfer of powder and reconstitution fluid into the metering device for medical use. In some implementations, the metering device comprises a housing, containing a metering chamber that defines a volume. The housing also has a connection portion, an extraction portion, and a metering member in fluid communication with the metering chamber. In other embodiments, the metering device additionally includes a dispensing aid that facilitates the dispensing of powder from a first container into the metering chamber through the connection portion. The connection portion is capable of insertion into a second container that contains fluid, thereby allowing a mixture of powder and fluid to form in the metering chamber. The mixture, then ready for use, is removable from the metering chamber through the extraction portion by an extraction device.

Abstract: Various embodiments of medical detector systems as well as their methods of operation are disclosed. In one embodiment, one or more detectors are coupled to wearable structures for detecting at least a first tracer within a body portion. In another embodiment, one or more detectors are coupled to a wearable structure, where the detector corresponds to a CMOS chip that directly detects a first radioactive tracer.

Abstract: The present invention relates to compounds of formula I: and pharmaceutically acceptable salts thereof, pharmaceutical compositions comprising the compounds of formula I, and methods of using the compounds, salts and compositions in the treatment of various disorders associated with CRM1 activity.

Abstract: This invention relates to alpha-4 binding antibodies, and fragments thereof.

Abstract: Antibodies that specifically bind to VLA-1 integrin and methods of using these antibodies to treat immunological disorders in a subject. Also included are crystal structures of complexes formed by VLA-1 antibodies and their ligands, and VLA-1 antagonists and agonists identified by using the structure coordinates of these structures.

Abstract: Methods of treating a subject having a condition characterized by at least one of neurodegeneration and neuroin-flammation are provided. Methods of reducing astrogliosis in a subject having a condition characterized by increased astrogliosis are also provided. Methods of providing neuroprotection to a subject in need thereof are also provided.

Abstract: The invention provided herein includes isolated polypeptides that specifically block the interaction between neonatal Fc receptor (FcRn) and albumin. Blocking the interaction treats diseases and conditions caused by increased amounts of albumin or modified albumin that possesses pathogenic properties wherein it is deemed desirable to decrease albumin levels. Accordingly, also provided are methods of using these isolated polypeptides to treat various diseases and conditions caused by increased amounts of albumin or modified albumin that possesses pathogenic properties. The invention provided herein also includes isolated polypeptides capable of binding to a non-IgG and non-albumin competitive site on an FcRn alpha 3 domain. These can be useful for tracking FcRn without inhibiting IgG or albumin binding or function. Accordingly, the invention also includes methods and systems to track FcRn without inhibiting IgG or albumin binding or function.