Abstract: Aspects of the invention are directed to arginine-free polypeptide-containing compositions and methods for treating disorders associated with inflammation or the autoimmune response. In particular, the polypeptide is etanercept.

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: Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of MECP2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of MECP2. Methods for modulating expression of MECP2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of MECP2.

Abstract: Provided are compounds of Formula I, or pharmaceutically acceptable salts thereof, and methods for their use and production.

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 are compounds of Formula (I), or pharmaceutically acceptable salts thereof, and methods for their use and production.

Abstract: Provided herein are methods of treating multiple sclerosis with a fumarate, wherein the fumarate is a dialkyl fumarate, a monoalkyl fumarate, a combination of a dialkyl fumarate and a monoalkyl fumarate, a prodrug of monoalkyl fumarate, a deuterated form of any of the foregoing, or a pharmaceutically acceptable salt, clathrate, solvate, tautomer, or stereoisomer of any of the foregoing, or a combination of any of the foregoing. The methods provided herein improve the safety of treatment by informing and monitoring patients undergoing treatment regarding progressive multifocal leukoencephalopathy, and/or by monitoring lymphocyte count.

Abstract: The invention relates to methods of assessing a patient's risk of developing Progressive Multifocal Leukoencephalopathy (PML).

Abstract: A stable aqueous pharmaceutical formulation comprising a therapeutically effective amount of an antibody, polysorbate 80, a buffer which inhibits polysorbate oxidation is described along with methods of making the preparation. Also described are formulations with high antibody concentrations which maintain fixed volumes and which may be used on patients of variable weight.

Abstract: Methods and apparatuses for dispersing a compound within the patient are described. In one embodiment, an effective amount of a compound may be present in the cerebrospinal fluid of a patient and a force may be applied to the torso of the patient sufficient to enhance the convection of the cerebrospinal fluid. In another embodiment, a bolus including a compound may be injected into the cerebrospinal fluid of a patient. The volume of the bolus may be between about 5% and 30% of the total volume of the cerebrospinal fluid of the patient.

Abstract: The invention herein describes a method to purify a target oligonucleotide using hydrophobic interaction chromatography (HIC). The method includes adding a salt to a mixture of the target oligonucleotide and product-related impurities, applying the diluted mixture, at a particular dynamic loading capacity, to the hydrophobic interaction chromatography resin (or hydrophobic adsorbent), washing the hydrophobic adsorbent with an aqueous salt solution, eluting the target oligonucleotide with a eluting solution, and collecting the eluent comprising the target oligonucleotide.

Abstract: Disclosed herein are antisense compounds and methods for decreasing Tau mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Tau-associated diseases, disorders, and conditions.

Abstract: Provided herein are compositions and methods for facilitating or enhancing delivery of nucleic acids, such as synthetic mRNAs, into cells or tissues. Such compositions and methods may include use of a targeting moiety-conjugated, such as an N-acetylgalactosamine (GalNAc)-conjugated, oligonucleotide to facilitate or enhance delivery.

Abstract: Compounds of formula I: and pharmaceutically acceptable salts, solvates, or hydrates thereof, pharmaceutical compositions comprising the compounds of formula I, and methods of using the compounds, salts, solvates, or hydrates and the compositions in the treatment of various disorders associated with CRM1 activity.

Abstract: A sandwich wrap including a flexible, edible sheet that is adapted to enfold an edible filling whereby the resulting enclosing tube has both ends open and an auxiliary support situated under the lowermost open end when the wrap is held vertically for consumption, thus restraining filling from exiting. A method using an assembly frame positions the auxiliary support. Filling is stocked in a capped filling storage tube including at least two components that can be disassembled from inside the edible enclosing tube to give the edible enclosed filling. The food product may include a set of separately enclosed different fillings and each edible enclosing tube may be shaped around the filling storage tube to give a cross-sectional form of a circle sector so that the set forms a right circular cylinder. A seepage container assists assembly in advance of an order to avoid impairment by moisture seeping out of fillings.

Abstract: The present disclosure describes novel hybrid soluble ActRIIB-ECD polypeptides which fully retain binding affinity for myostatin and activin A but demonstrate significantly reduced binding to BMPs, especially BMP-9. The novel compositions described herein can be used to prepare novel hybrid ActRIIB ligand trap proteins, which can be used for modulating the growth of muscle, bone, cartilage, fat, fibroblast, blood and neuronal tissue to counteract muscle wasting, bone loss, anemia, inflammation and fibrosis in a therapeutically meaningful manner. Because these novel next-generation myostatin/activin inhibitors are safer and more effective molecules than the currently available myostatin inhibitors, they are useful in a wide variety of clinical indications.

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject.

Abstract: Provided are compounds of Formula (I), or pharmaceutically acceptable salts thereof, and methods for their use and production.

Abstract: The present invention provides compounds of formula (I) or (II) or a pharmaceutically acceptable salt thereof and their use in modulating the activity of S1P) receptors. Also provided is a pharmaceutical composition comprising the compounds of formula (I) or (II) or a pharmaceutically acceptable salt thereof and a pharmaceutically acceptable excipient or carrier.