The present invention features compositions and methods useful in inhibiting the expression of NFIX within a cell and thereby treating patients suffering from a hemoglobinopathy such as sickle cell disease or β-thalassemia

Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating, preventing, or ameliorating hypercholesterolemia and/or atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.

Short-acting Factor VII peptides are disclosed. A shortened half-life is desirable for treatment of acute bleeding and similar disorders. Modification of the sialylation and/or glycosylation of Factor VII and variants thereof produced peptides useful in treating conditions of acute bleeding.

Provide herein are compounds and pharmaceutical compositions suitable as modulators of hemoglobin, methods and intermediates for their preparation, and methods for their use in treating disorders mediated by hemoglobin and disorders that would benefit from tissue and/or cellular oxygenation.

The invention provides anti-cluster of differentiation 3 (CD3) antibodies and methods of using the same.

Disclosed embodiments concern novel interleukin receptor associated kinases (IRAK) inhibitors and compositions comprising such inhibitors. Also disclosed are methods of making and using the compounds and compositions. The disclosed compounds and/or compositions may be used to treat or prevent an IRAK-associated disease or condition.

The present application provides a radiation damage protecting agent comprising hydrogen gas as an active ingredient at a concentration of 18.5% by volume or less, for treating or alleviating, in a hyperbaric capsule under a pressure higher than standard atmospheric pressure, radiation damage in a human patient who has been exposed to radiation or who has received or receives radiotherapy, and a hyperbaric capsule for administering a hydrogen gas-containing therapeutic agent such as the radiation damage protecting agent to a patient including a human.

The present application provides a radiation damage protecting agent comprising hydrogen gas as an active ingredient at a concentration of 18.5% by volume or less, for treating or alleviating, in a hyperbaric capsule under a pressure higher than standard atmospheric pressure, radiation damage in a human patient who has been exposed to radiation or who has received or receives radiotherapy, and a hyperbaric capsule for administering a hydrogen gas-containing therapeutic agent such as the radiation damage protecting agent to a patient including a human.

Compositions comprising microbiome regulators are provided, as well as methods of using the same for the modulation of the human microbiota and to treat or prevent related diseases, disorders, or conditions.

The present disclosure provides methods for treating, preventing, or inhibiting diseases in patients having one or more mutations in complement factor H (CFH), complement component 3 (C3), and complement factor B (CFB). Also provided are combination therapies comprising a CFH protein and a VEGF antagonist for treating neovascularization-associated ocular diseases.