Abstract: The invention relates to Cebranopadol for use in the treatment of pain, wherein Cebranopadol is administered according to an administration regimen comprising (i) a first administration interval, which lasts for at least 2 consecutive days, wherein a first daily dose of Cebranopadol is administered on every day of the first administration interval; and (ii) a second administration interval, which lasts for at least 2 consecutive days and directly follows the first administration interval without interruption, wherein a second daily dose of Cebranopadol is administered on every day of the second administration interval; wherein the first daily dose of Cebranopadol is lower than the second daily dose of Cebranopadol.

Abstract: The present description relates' to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.

Abstract: A fusion molecule having phagocytosis-inducing activity is disclosed. The fusion molecule contains a first region capable of binding a TAM receptor and a second region capable of binding to a target substance of which aberrant accumulation is associated with or characteristic of diseases. The fusion molecule effectively clears and/or reduces and/or suppresses accumulated abnormal proteins, such as beta-amyloid, tau, alpha-synuclein, huntingtin, or prion, or the like. Uses of the fusion molecule are disclosed. The fusion molecule can be used for prevention or treatment of proteinosis caused by the abnormal accumulation of substances.

Abstract: Provided herein are inhibitors of TNF?, pharmaceutical compositions comprising the inhibitory compounds, and methods for using the TNF? inhibitory compounds for the treatment of diseases or disorders.

Abstract: Provided herein are proteins, which are capable of being transported across the blood-brain barrier (BBB) and comprise an acid alpha-glucosidase (GAA) enzyme-Fc fusion polypeptide. Certain embodiments also provide methods of using such proteins to treat Pompe disease.

Abstract: The disclosure is directed in part to variant capsid polypeptides that can be used to deliver payloads.

Abstract: The present disclosure relates to novel compounds, to said compounds for use as a medicine, more in particular for the prevention or treatment of diseases mediated by activity of YAP/TAZ-TEAD transcription, yet more in particular for the prevention or treatment of cancer or fibrosis. The present disclosure also relates to a method for the prevention or treatment of said diseases comprising the use of the novel compounds.

Abstract: The present invention relates to a composition for hair improvement, and more specifically to a composition for promoting hair growth, or preventing or treating hair loss, comprising a guanine derivative having no toxicity as an active ingredient. The composition has excellent effects of promoting hair growth and regenerating hair follicles, and thus can be effectively used in various fields such as pharmaceutical, food, feed, and cosmetic compositions for promoting hair growth, or preventing, treating, and improving hair loss.

Abstract: Disclosed herein are pyridyl compounds. Also described are specific conjugated nicorandil compounds. Also disclosed are pharmaceutical compositions that include the compounds. Methods of using the pyridyl compounds are disclosed for the treatment of diseases or conditions related to kidney or kidney functions.

Abstract: This disclosure relates to BTK inhibitor compounds for treating or preventing cancer by daily oral dosing in human subjects in need thereof. The description also provides BTK inhibitor compounds for degrading BTK in vivo by daily oral dosing in human subjects in need thereof.

Abstract: Provided herein are proteins, which are capable of being transported across the blood-brain barrier (BBB) and comprise an acid alpha-glucosidase (GAA) enzyme-Fc fusion polypeptide. Certain embodiments also provide methods of using such proteins to treat Pompe disease.

Abstract: Provided herein are compounds, pharmaceutical compositions comprising the compounds, methods of preparing the compounds, and methods of using the compounds and compositions in treating diseases or disorders in a subject where the subject is in need of an inhibitor of MEK where the compound is according to formula (I): where R1, R2, R2a, R3, R3a, R3b, and subscript n are as described herein.

Abstract: Multi-specific binding proteins that bind NKG2D receptor, CD 16, and a tumor-associated antigen selected from c-MET, KIT, F3, IGF1R, Lewis Y, MUC13, MUC4, MCAM, LRRC32, sialyl-Tn, gpA33, GD3, GM2, EPHA3, TNFRSF10A, TNFSF11, CD74, and PMEL are described, as well as pharmaceutical compositions and therapeutic methods useful for the treatment of cancer.

Abstract: The present invention relates to combination therapies for treating KRas G12C cancers. In particular, the present invention relates to methods of treating cancer in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of a combination of a SHP-2 inhibitor and a KRAS G12C inhibitor of Formula (I), Formula I-A or Formula I-B, pharmaceutical compositions comprising a therapeutically effective amounts of the inhibitors, kits comprising the compositions and methods of use therefor.

Abstract: The present invention is directed to novel translational inhibitors that bind covalently with eukaryotic initiation factor 4E (eIF4E) and inhibit or modulate the activity of eIF4E, as well as stereoisomers, tautomers and pharmaceutically acceptable salts of such compounds. The present invention also is directed to pharmaceutically acceptable compositions containing such translational inhibitors and associated methods for treating conditions that would benefit from eIF4E inhibition including, but not limited to, treatment of inflammation and various cancers.

Abstract: The present invention relates to pharmaceutical compounds of Formula (I): as further described herein, and pharmaceutical compositions containing such compounds, as well as methods of using these compounds to modulate the activity of aryl hydrocarbon receptor (AhR) and to treat conditions comprising inflammation inside the intestinal tract and certain disorders of the central nervous system affected by the gut-brain axis, including IBD, ulcerative colitis, Crohn's disease, Huntington's disease, and multiple sclerosis.

Abstract: Disclosed herein are compositions comprising an NSAID such as meloxicam and/or rizatriptan in combination with a cyclodextrin and/or a carbonate or a bicarbonate. These compositions may be orally administered, for example, to improve the bioavailability or pharmacokinetics of the NSAID for the treatment of pain such as migraine, arthritis, and other conditions. Also disclosed herein are methods of treating pain, such as migraine, comprising administering meloxicam and rizatriptan to a human being suffering from pain, such as migraine. For migraine, these methods may be particularly useful when the meloxicam and rizatriptan are administered while the human being is suffering from an acute attack of migraine pain or migraine aura. In some embodiments, the combination of meloxicam and rizatriptan may be administered in a manner that results in a Tmax of meloxicam of 3 hours or less.

Abstract: The present invention relates to a process for the preparation of a compound of Formula (G), comprising reacting a compound of Formula (H) with a compound of Formula (D) in the presence of a palladium catalyst to afford the compound of Formula (G).

Abstract: The present disclosure provides methods of administering chimeric and hybrid Factor VIII (FVIII) polypeptides comprising FVIII and Fc to subjects at risk of developing inhibitory FVIII immune responses, including anti-FVIII antibodies and/or cell-mediated immunity. The administration is sufficient to promote coagulation and to induce immune tolerance to FVIII. The chimeric polypeptide can comprise full-length FVIII or a FVIII polypeptide containing a deletion, e.g., a full or partial deletion of the B domain.

Abstract: The present invention relates to methods, compositions, and combinations for inhibiting the NLRP3 inflammasome and/or LON protease as well as methods, compositions, and combinations for treating cancer, particularly blood cancer or brain cancer. In certain aspects, the compositions and combinations include a synthetic triterpenoid, such as 1-[2-Cyano-3,12-dioxooleana-1,9(11)-dien-28-oyl]-4(-pyridin-2-yl)-1H-imidazole (“CDDO-2P-Im”) and 1-[2-Cyano-3,12-dioxooleana-1,9(11)-dien-28-oyl]-4(-pyridin-3-yl)-1H-imidazole (“CDDO-3P-Im”).