摘要:
Disclosed herein are α7β1 integrin modulatory agents and methods of using such to treat conditions associated with decreased α7β1 integrin expression or activity, including muscular dystrophy. In one example, methods for treating a subject with muscular dystrophy are disclosed. The methods include administering an effective amount of an α7β1 integrin modulatory agent to the subject with muscular dystrophy, wherein the α7β1 integrin modulatory agent increases α7β1 integrin expression or activity as compared to α7β1 integrin expression or activity prior to treatment, thereby treating the subject with muscular dystrophy. Also disclosed are methods of enhancing muscle regeneration, repair, or maintenance in a subject and methods of enhancing α7β1 integrin expression by use of the disclosed α7β1 integrin modulatory agents. Methods of prospectively preventing or reducing muscle injury or damage in a subject are also disclosed.
摘要:
Urea-based and bis-urea based compounds and analogues thereof are disclosed. These compounds are useful in the treatment of androgen-dependent diseases or disorders and androgen receptor-mediated diseases or disorders. Specifically, the compounds are useful in the treatment of diseases or disorders that are AR negative.
摘要:
The invention relates to a pharmaceutical formulation or composition intended to be administered orally in the form of a tablet, a coated tablet or a capsule, for the prevention or treatment of intestinal disorders, such as irritable bowel syndrome, comprising an intestinal motility modifier, an agent that prevents gas retention, digestive enzymes, a binding agent, a diluent, an adsorbent agent, a disintegrating agent, a lubricant and a glidant, effective for normalising colon transit, for providing an analgesic activity, for providing an antispasmodic activity and for reducing symptoms related to intestinal gas, such as distention, abdominal pain and flatulence.
摘要:
The present invention features compositions comprising a plurality of therapeutic agents wherein the presence of one therapeutic agent enhances the properties of at least one other therapeutic agent. In one embodiment, the therapeutic agents are cystic fibrosis transmembrane conductance regulators (CFTR) such as a CFTR corrector or CFTR potentiator for the treatment of CFTR mediated diseases such as cystic fibrosis. Methods and kits thereof are also disclosed.
摘要:
The embodiments include methods of treating conditions requiring removal or destruction of cellular elements, such as benign or malignant tumors using compounds based on small peptides in combination with additional active agent(s). The method includes, but is not limited to, administering the compounds intramuscularly, orally, intravenously, intrathecally, intraprostatically, intratumorally, intranasally, topically, transdermally, etc., either alone or conjugated to a carrier to a mammal in need thereof.
摘要:
Provided are methods for eliminating hematologic cancer stem cells in vivo, and thus preventing cancer relapse. The methods comprise a Signal Transducer and Activator of Transcription 5 (STAT5) antagonist (e.g., a PPARγ agonist) after the patient has had ana initial course of treatment with an anti-cancer agent (e.g., a TKI), to eliminate residual cancer stem cells which cause relapse.
摘要:
This invention provides novel indole, indazole, benzimidazole, indoline, quinolone, isoquinoline, and carbazole selective androgen receptor degrader (SARD) compounds, pharmaceutical compositions and uses thereof in treating prostate cancer, advanced prostate cancer, castration resistant prostate cancer, androgenic alopecia or other hyper androgenic dermal diseases, Kennedy's disease, amyotrophic lateral sclerosis (ALS), and uterine fibroids, and to methods for reducing the levels of androgen receptor-full length (AR-FL) including pathogenic and/or resistance mutations, AR-splice variants (AR-SV), and pathogenic polyglutamine (polyQ) polymorphisms of AR in a subject.
摘要:
The disclosed subject matter provides N- substituted hydroxylamine derivative compounds, pharmaceutical compositions and kits comprising such compounds, and methods of using such compounds or pharmaceutical compositions. In particular, the disclosed subject matter provides methods of using such compounds or pharmaceutical compositions for treating heart failure.
摘要:
There is provided herein a nanovesicle comprising a monolayer of phospholipid, porphyrin-phospholipid conjugate and a peptide encapsulating a hydrophobic core, wherein the peptide comprises an amino acid sequence capable of forming at least one amphipathic α-helix; the porphyrin-phospholipid conjugate comprises one porphyrin, porphyrin derivative or porphyrin analog covalently attached to a lipid side chain, preferably at the sn-1 or the sn-2 position, of one phospholipid; the molar % of porphyrin-phospholipid conjugate to phospholipid is 35% or less; the nanovesicle is 35 nm in diameter or less.