Biogen MA Inc.

A burr hole device is configured to receive a catheter and a cable of a sheath. The sheath is configured to receive a portion of the catheter and has an electrode. When the catheter and sheath are implanted with the burr hole device, the catheter and electrode of the sheath are implanted in a brain. Systems and apparatuses may include the burr hole device and/or a cranial port device, the catheter, and the sheath.

Featured are biomarkers for progressive multifocal leukoencephalopathy (PML). Also provided are methods for reducing the occurrence or severity of PML during immunomodulator therapy or identification of PML in an immunocompromised subject.

Among other things, the present disclosure provides compositions, e.g., isolated nucleic acids, vectors, and recombinant adeno-associated viruses (rAAVs), comprising a nucleic acid sequence encoding a PRPF31 polypeptide. In some embodiments, the present disclosure provides methods of increasing expression of PRPF31, e.g., in a subject. In some embodiments, the present disclosure provides methods of treating retinal degeneration in a subject. In some embodiments, the present disclosure provides methods of treating retinitis pigmentosa (RP) in a subject.

Aspects of the application relate to compositions and methods for treating spinal muscular atrophy in a subject. In particular, this application provides therapeutic combinations of a recombinant nucleic acid that encodes the survival of motor neuron 1 (SMN1) protein (e.g., in a viral vector), and an antisense oligonucleotide (ASO) that increases full-length survival of motor neuron 2 (SMN2) mRNA (e.g., that is targeted to a nucleic acid molecule encoding the survival of motor neuron 2 (SMN2) and that promotes the inclusion of exon 7 in SMN2 mRNA).

Natalizumab is a safe and efficacious treatment for inflammatory and autoimmune diseases, such as multiple sclerosis, Crohn's Disease, and rheumatoid arthritis. Chain swapping between natalizumab and IgG4 molecules acts to reduce the level of bivalent natalizumab present following administration of natalizumab, and thus to lower the activity of natalizumab in the patient. Differences in IgG4 levels across patients or within a single patient across time may change the pharmacokinetic profile of natalizumab. Patients with lower levels of IgG4 may experience higher nadir levels of natalizumab during a dosing period. Monitoring IgG4 and/or bivalent natalizumab levels, and determining a dose or dosage period based on the monitoring may improve the safety and/or efficacy of natalizumab therapy.

A burr hole device is configured to receive a catheter and a cable of a sheath. The sheath is configured to receive a portion of the catheter and has an electrode. When the catheter and sheath are implanted with the burr hole device, the catheter and electrode of the sheath are implanted in a brain. Systems and apparatuses may include the burr hole device and/or a cranial port device, the catheter, and the sheath.

The present disclosure provides compounds of Formula I, and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled compounds, and prodrugs thereof. The provided compounds may be glycogen synthase kinase 3 (GSK3) inhibitors. The present disclosure also provides pharmaceutical compositions, combination therapies, and kits comprising the compounds, and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled compounds, or prodrugs thereof, and methods of treating or preventing diseases and disorders associated with GSK3.

A system includes a first port comprising a first inlet, a first outlet, a first fluid pathway extending from the first inlet to the first outlet, a second inlet, a second outlet, and a second fluid pathway extending from the second inlet to the second outlet. The system further includes one or more CSF catheters having a first lumen, a first distal opening in fluid communication with the first lumen, a second lumen, and a second distal opening in fluid communication with the second lumen. The one or more CSF catheters are, or are configured to be, operatively coupled with the first implantable device such that the first lumen is in fluid communication with the first fluid pathway and the second lumen is in fluid communication with the second fluid pathway. At least the first distal opening is configured to be placed in the CSF-containing space. The system further includes a second port having a third inlet, a third outlet, and a third fluid pathway extending from the third inlet to the third outlet. The system also includes a port catheter configured to operatively couple the third fluid pathway to the second fluid pathway.

The invention provides a composition comprising an anti-tumor necrosis factor (TNF)-alpha polypeptide, wherein the composition produces a reduced level of immunogenicity when administered to a subject, as well as a treatment method involving the administration of the composition.

The present disclosure provides a compound of Formula (I′), or a pharmaceutically acceptable salt thereof and its use in, e.g. treating a condition, disease, or disorder in which lowering mutant huntingtin protein (“mHTT”) in a subject is of therapeutic benefit, specifically in treating Huntington disease (“HD”). This disclosure also features a composition containing the same as well as methods of using and making the same.