68 results about "Blood Disorder" patented technology

The invention relates to compounds of formula (Ia) and to their use in treating or preventing an inflammatory disease, a disease associated with an undesirable immune response, cancer, obesity, a diabetic disease or a blood disorder:wherein RA, RB, RC and RD, X, m and n are as defined herein.

The invention relates to a compound of formula (I)which is 6-((1H-pyrazol-4-yl)sulfonyl)-2-((1-cyclopropyl-1H-pyrazol-3-yl)methyl)phthalazin-1(2H)-one; or a salt and / or solvate thereof;and to its use in treating or preventing an inflammatory disease, a disease associated with an undesirable immune response, cancer, obesity, a diabetic disease or a blood disorder.

The present invention relates to a solid nutraceutical composition for the prevention and treatment of cognitive disorders, consisting of an emulsion and a mixture of seed and oil, all of which are naturally derived. The emulsion contains plukenetia volubilis oil; an extract of plant origin, such as turmeric, pepper or cocoa; an emulsifier selected from lecithin, saponaria or maguey extract, or egg white; the sweetening agent is selected from fructo-oligosaccharide, stevia rebaudiana or maguey extract, Italian bee honey and cane sugar; and air is contained. Seeds and oil include pumpkin seeds and plukenetia volubilis oil. Other ingredients selected from cocoa powder, coconut oil, fruit or seed mass, natural juice and protein are also added. Thus, the nutraceutical comprises 3 to 5 sources of fatty acids, phytosterols and triacylglycerol, 2 to 8 sources of antioxidants, up to 3 sources of hypoglycemic agents, and up to 4 sources of anti-cholesterol and anti-inflammatory compounds. In addition, the nutritional health-care product also contains proteins, vitamins, macroelements, microelements and substances for promoting chewing and swallowing of subjects and patients suffering from cognitive impairment. The nutritional health-care product is suitable for preventing and treating Alzheimer's disease; mental, behavioral and developmental disorders, including depression; neurological, digestive, immune, endocrine and circulatory disorders; cardiovascular, cerebrovascular, infectious, parasitic, neoplastic and blood diseases; fatigue, fatigue and malnutrition.

Provided herein are antibodies that bind to von Willebrand factor to an epitope positioned at a cysteine knot (CK) domain. Such antibodies are useful for methods of treatment, such methods of treating one or more blood disorders characterized by excessive bleeding.

The application discloses a blood disease progress prediction method and system based on a knowledge graph, which comprises the following steps: constructing a blood disease knowledge graph based on blood disease medical knowledge and clinical data; collecting target patient data and generating a time series medical data set; mapping the time series medical data set to the knowledge graph to generate a disease evolution subgraph; encoding, propagating and aggregating nodes and edges in the disease evolution subgraph through R-GCN to generate a graph representation vector; screening, normalizing and time series splicing the graph representation vector to generate a multi-modal input sequence; inputting the multi-modal input sequence into an improved FEDformer model to output a blood disease prediction result; and generating a blood disease progress prediction conclusion and a risk warning signal according to the blood disease prediction result. The application combines a graph neural network with a time series prediction model, thereby improving the accuracy and real-time performance of blood disease progress prediction.

Disclosed are novel compounds that are antagonists of the human GPR39 protein. Disclosed are pharmaceutical compositions of antagonists of the human GPR39 protein and methods of use. In particular, disclosed herein are methods of using the antagonists to treat diseases or conditions, including cardiovascular conditions, endocrine system and hormone disorders, cancer disorders, metabolic diseases, gastrointestinal and liver diseases, blood disorders, neurological disorders, and respiratory diseases.

Described herein are compounds that activate pyruvate kinase, pharmaceutical compositions, and methods of using the same. The compounds are represented by Formula (I), wherein R 1 , R 2 , R a , R b , R j , R k , and Q are as defined herein.

Lentiviral vector (LV) formulations, and pharmaceutical compositions comprising such LV formulations, with improved stability and suitable for systemic administration are provided. Methods for treating disorders, especially blood disorders, using systemic administration of LV formulations are also provided.

Lentiviral vector (LV) formulations, and pharmaceutical compositions comprising such LV formulations, with improved stability and suitable for systemic administration are provided. Methods for treating disorders, especially blood disorders, using systemic administration of LV formulations are also provided.

Inhibitory nucleic acid molecules are provided as well as methods of use thereof. Specifically, the disclose provides an Inhibin BetaA (inhba) inhibitory nucleic acid molecule, which is an antisense oligonucleotide, siRNA, or shRNA. Further disclosed are sequences that are targeted by the inhba inhibitory nucleic acid molecule; and methods of treating a disease or disorder in a subject comprising administering an inhba inhibitory nucleic acid molecule to the subject, wherein the disease or disorder is selected from the group consisting of cancers, autoimmune diseases, fibrotic disorders, blood disorders, allergies and allergic diseases, heart failure, neurodegenerative diseases, cachexia and inflammatory diseases.

Provided herein are compounds and compositions thereof for activating nuclear factor erythroid 2-related factor 2 (Nrf2) for treating red blood cell diseases and inflammatory diseases.

The present invention relates to nicotinamide mononucleotide derivatives of formula (I) for use in the treatment and / or prevention of blood disorders, in particular sickle cell disease. The invention also relates to a pharmaceutical composition comprising the compound of formula (I) for use in the treatment and / or prevention of erythrocyte disorders, in particular sickle cell disease.

The present invention relates to a compound of formula (I) which is 6-((1H-pyrazol-4-yl)sulfonyl)-2-((1-cyclopropyl-1H-pyrazol-3-yl)methyl)phthalazin-1(2H)-one [Formula 1] or a salt and / or solvate thereof; and its use in the treatment or prevention of an inflammatory disease, a disease associated with an unwanted immune response, cancer, obesity, a diabetic disease, or a blood disorder.

The present invention relates to a nicotinamide mononucleotide derivative of formula (I) which is used for treating and / or preventing blood disorders, especially sickle cell disease. The present invention also relates to a pharmaceutical composition comprising a compound of formula (I) which is used for treating and / or preventing red blood cell disorders, especially sickle cell disease.

In accordance with various embodiments of the present disclosure, a method for non-invasive blood disorder monitoring is provided. In some embodiments, the method comprises emitting light from a photonic integrated circuit-scale dual frequency comb at a plurality of different wavelengths via a hand-held device directed at one or more bodily structures of a patient having been diagnosed with a blood disorder; detecting acoustic waves from thermo-elastic changes in red blood cells within the bodily structures; generating an optical absorption spectrum from the detected acoustic waves; identifying elements within the bodily structures exposed to the emitted light based on the optical absorption spectrum; and generating a three-dimensional image of one or more blood vessels based on the optical absorption spectrum from the detected acoustic waves from the red blood cells to identify a blockage or partial blockage of blood flow in the one or more blood vessels.

Compositions and methods for treating a blood disorder in a subject comprising delivering a nucleic acid molecule including a nucleotide sequence encoding two to six guide RNAs (gRNAs) into a hematopoietic stem cell (HSC), a hematopoietic progenitor cell (HPC), or a population of hematopoietic stem and progenitor cells (HSPCs) are described.

Devices and methods for reducing or limiting bleeding in an animal by stimulating the spleen with focused ultrasound (FUS). The devices and methods can be used to treat blood disorders such as hemophilia, or to reduce bleeding during surgery or due to trauma. The methods can be administered to a patient non-invasively by applying ultrasound energy transcutaneously.

The disclosure provides method for treating clonal hematopoiesis disorders such as clonal hematopoiesis, myelodysplasia, and leukemia.

The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta-hemoglobinopathies), such as sickle cell disease and beta-thalassemia.

Provided herein are antibodies, compositions, and methods of use thereof. The disclosed antibodies bind to von Willebrand factor in blood plasma of a subject. Such antibodies are useful for methods of treatment, such methods of treating one or more blood disorders characterized by excessive bleeding. Also provided herein are methods of treating blood disorders by administering a combination of an anti-VWF antibody and von Willebrand Factor.

The present invention relates to an integration-defective lentiviral vector (IDLV) comprising a nucleic acid, the said nucleic acid comprising, between a 5′ LTR sequence and a 3′ LTR sequence, at least one nucleus export signaling sequence; at least one nucleic acid sequence of interest; and at least one nuclease site. The invention further relates to an isolated cell comprising said IDLV, a pharmaceutical composition comprising said IDLV or said isolated cell, and their pharmaceutical use in the treatment of a disease selected from the group consisting of immune diseases, viral infections, tumors and blood diseases; and / or a disease caused by the lack of a protein or by the presence of an aberrant non-functional one in an individual in need thereof.

The present invention provides a rapid, non-invasive, and antibody-free method for identifying AA and MDS based on Raman spectroscopy. This method utilizes the peak intensity of Raman spectra as a biomarker for the preparation of products for predicting or detecting blood disorders. The specificity and significant Raman shifts of the present invention are valuable biomarkers for early disease identification, improving diagnostic efficiency, reducing testing costs, and promoting the rapid and accurate diagnosis of AA and MDS.

The application relates to the application of YAP protein in the proliferation or apoptosis of vascular smooth muscle cells under the stimulation of mechanical stress. The application relates to the correlation research of YAP protein and the Hippo pathway, and also relates to YAP inhibitors including siRNA and shRNA. After the vascular smooth muscle cells are transfected by the YAP inhibitors (siRNA and shRNA), the expression of YAP is knocked down. When YAP is knocked down, the cell proliferation of the vascular smooth muscle cells under the stimulation of mechanical stress is inhibited. The application opens up a new research prospect for the treatment of blood diseases, and provides a new idea for the deep development of the YAP inhibitors and the clinical treatment of blood diseases.

The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta-hemoglobinopathies), such as sickle cell disease and beta-thalassemia.

Disclosed herein are novel compounds that are antagonists of the human GPR39 protein. Pharmaceutical compositions and methods of use of antagonists of human GPR39 protein are disclosed. In particular, disclosed herein are methods of treating diseases or conditions, including cardiovascular conditions, endocrine system and hormone disorders, cancer disorders, metabolic disorders, gastrointestinal and liver disorders, hematological disorders, neurological disorders, and respiratory disorders, using the antagonists.

The present disclosure includes compositions possessing a recombinant H-ferritin homopolymer that has a plurality of H-ferritin subunits with an amino acid sequence of SEQ ID NO: 1. Methods for treating an iron-related condition in a subject and for treating a neurological or blood disorder in a subject with such compositions are also disclosed.