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

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: 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: 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.

Abstract: Antibodies and antibody fragments that specifically bind to glycoprotein IIb/IIIa (GPIIb/IIIa) are disclosed. Chimeric molecules comprising such antibodies or antigen-binding fragments are also disclosed. In addition, methods of using the disclosed antibodies, antibody fragments, and chimeric molecules, e.g., to target agents to platelets and for the treatment or prevention of diseases or disorders are provided.

Abstract: A process for reducing the environmental contaminants in a ISO 8217 compliant Feedstock Heavy Marine Fuel Oil, the process involving: mixing a quantity of the Feedstock Heavy Marine Fuel Oil with a quantity of Activating Gas mixture to give a feedstock mixture; contacting the feedstock mixture with one or more catalysts to form a Process Mixture from the feedstock mixture; separating the Product Heavy Marine Fuel Oil liquid components of the Process Mixture from the gaseous components and by-product hydrocarbon components of the Process Mixture and, discharging the Product Heavy Marine Fuel Oil. The Product Heavy Marine Fuel Oil is compliant with ISO 8217 for residual marine fuel oils and the sulfur and Specific Contaminants have concentration less than 0.5 wt %., wherein the Specific Contaminates are selected from the group consisting of: vanadium, sodium, aluminum, silicon, calcium, zinc, phosphorus, nickel, iron and combinations thereof.

Abstract: Aspect of the disclosure relate to methods of assessing a bioreactor culture that involve determining a culture parameter of the manufacturing-scale bioreactor culture using a model that relates a Raman spectrum to the culture parameter. Related bioreactor system are also provided.

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.

Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.

Abstract: The present invention pertains to a cell culture medium comprising manganese as a media supplement, which was shown to control recombinant protein glycosylation and methods of using thereof. The present invention further pertains to a method of controlling or manipulating glycosylation of a recombinant protein of interest in a large scale cell culture.

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.