Abstract: The present disclosure describes a convergent liquid phase process for manufacturing oligonucleotides by coupling two or more oligonucleotide fragments, each of which have two or more nucleotides. Also provided by the present disclosure are reaction steps involved the convergent liquid phase process.

Abstract: An intraventricular access device for infusion of drugs and aspiration of cerebrospinal fluid (CSF). The device includes a container configured to couple to an aspiration lumen through which CSF may be aspirated or through which drugs may be injected. The container is further configured to couple to an infusion lumen and to a drug pump via a catheter. The container thus forms a pathway connectable to the catheter through which a drug from the drug pump may be infused to flow through the infusion lumen into the CSF. The container includes a volume that is accessible by an aspiration needle, for example through a material that reseals upon extraction of the needle. Moreover, the container is configured to prevent cross contamination between a first fluid in the aspiration lumen and a second fluid in the infusion lumen.

Abstract: The invention relates to methods and reagents for analyzing samples for the presence of JC virus antibodies.

Abstract: Described herein are compounds represented by formula (I), or a pharmaceutically acceptable salt thereof, pharmaceutical compositions comprising the same and methods of preparing and using the same. The variables Ar, X, R1, R3, R4, Y1, Y2, m, n, and p are as defined herein.

Abstract: Described herein are compounds represented by formulas (IA) or (IB) or a pharmaceutically acceptable salt thereof, pharmaceutical compositions comprising the same and methods of preparing and using the same. The variables R1, R3, R4, Y1, Y2, Ar, Z and n are as defined herein.

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

Abstract: A device that regulates, monitors, and provides data on the progress of a large-volume injector during its injection sequence. The device comprises an injector and a monitor assembly. The injector has a syringe having a plunger, and a biasing device that biases the plunger through the syringe. The monitor assembly comprises a resistance unit configured to resist movement of the plunger, a gear connected to the plunger by the resistance unit, and a control unit configured to monitor the position, and regulate the rotation, of the gear. Wherein linear movement of the plunger causes the gear to rotate so that the control unit generates information on the position of the plunger.

Abstract: An isolated protein comprising a mutant soluble activin II receptor (ActRIIA or ActRIIB) extracellular domain (Ac-tRIIA-ECD or ActRIIB-ECD), wherein said mutant soluble ActRIIA-ECD or ActRIIB-ECD comprises a mutation to remove the N-linked glycosylation site corresponding to position N18 of SEQ ID NO: 1.

Abstract: Described herein are compounds represented by formulas (IA) or (IB) or a pharmaceutically acceptable salt thereof, pharmaceutical compositions comprising the same and methods of preparing and using the same. The variables R1, R3, R4, Y1, Y2, Ar, Z and n are as defined herein.

Abstract: Provided are novel dipeptide repeat (DPR) specific antibodies (e.g., human-derived antibodies) as well as synthetic variants and biotechnological derivatives thereof, capable of binding C9orf72 poly-glycine-alanine DPRs, as well as methods and uses related thereto. The antibody of the present invention can be used in pharmaceutical and diagnostic compositions for DPR protein-targeted immunotherapy and diagnostics.

Abstract: Provided are compounds of Formula (I): or pharmaceutically acceptable salts thereof, wherein R1, R2, R3, R4, R5, R7, R8, R9, A1, A2, Q1, Q2, Q3, A, Z, m and n are as defined herein; pharmaceutical compositions comprising said compounds or pharmaceutically acceptable salts thereof, and pharmaceutically acceptable excipients; and methods of treating a disorder responsive to inhibition of Bruton's tyrosine kinase using said compounds, or pharmaceutically acceptable salts thereof, or said pharmaceutical compositions.

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

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

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 invention relates to anti-VLA-4 antibodies and binding fragments thereof. The invention further includes polynucleotides encoding said antibodies and binding fragments thereof and methods of manufacturing said antibodies and binding fragments thereof. The invention further includes methods of treating patients suffering from multiple sclerosis and/or epilepsy by administration of said antibodies and binding fragments thereof. The invention further includes methods of reducing the susceptibility to scrambling of a recombinant anti-alpha 4 antibody or a binding fragment thereof.

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

Abstract: The present invention relates to improved methods in the separation recombinant polypeptides with post-translational modifications from complex mixtures through the use of a cation exchange medium.

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: Provided are compounds of Formula (I): Formula (I), including compounds of Formulas (II), (III), (IV), (V) and (VI), wherein X, R1, R2, R3, R4 and n are as defined herein, and pharmaceutically acceptable salts thereof, and methods for their use and production. These compounds can be useful, e.g., in the treatment of disorders responsive to the inhibition of apoptosis signal-regulating kinase 1 (ASK1).