433 results about "Severe anaemia" patented technology

Aspects of the application provide hemojuvelin antagonists and methods of using the same in treating anemias of kidney disease and conditions associated with these anemias. Methods provided in the application relate to treatment of a subject having an anemia associated with chronic kidney disease and / or kidney disease in a subject that has a level of glomerular filtration rate lower than one or more thresholds.

The invention relates to the technical field of hemodialysis monitoring, and provides a hemodialysis on-line monitoring system and method, and the system comprises a data acquisition module which is used for collecting blood physiological parameters in a dialysis pipeline in real time; the blood component analysis module is connected with a blood loop of the dialysis machine and is used for detecting serum iron, ferritin and ferritin saturation indexes on line; and the data processing module is used for calculating the risk index of anemia, evaluating the iron utilization rate, constructing a model for measuring the iron deficiency erythropoiesis trend according to the collected and analyzed data, and training and optimizing the model. According to the method, individual parameters of patients are fused, personalized calculation is performed for iron metabolism rates and dialysis parameters of different patients, and data such as iron content, blood flow and dialysis fluid volume are acquired online in real time, so that iron concentration and infusion rate are accurately matched with real-time iron metabolism requirements of the patients, and anemia of the patients in the dialysis process is reduced.

The invention features polypeptides that include an extracellular ActRIIA variant. In some embodiments, a polypeptide of the invention includes an extracellular ActRIIA variant fused to an Fc domain monomer or moiety. The invention also features pharmaceutical compositions and methods of using the polypeptides to treat diseases and conditions involving low red blood cell levels, e.g., anemia or blood loss; fibrosis; or pulmonary hypertension.

The invention features polypeptides that include an extracellular ActRIIB variant. In some embodiments, a polypeptide of the invention includes an extracellular ActRIIB variant fused to an Fc domain monomer or moiety. The invention also features pharmaceutical compositions and methods of using the polypeptides to treat diseases and conditions involving weakness and atrophy of muscles, bone damage, low red blood cell levels (e.g., anemia or blood loss), fibrosis, and / or pulmonary hypertension.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., cold agglutinin disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupis nephritis (LN), bullous pemphigoid, Pemphigus, e.g., Pemphigus vulgaris (PV) and Pemphigus foliaceus (PF), and C3 glomerulopathy.

The present disclosure relates to compositions and methods of increasing levels of fetal hemoglobin (HbF) in cells. The present disclosure further relates to methods for treating patients suffering from blood cell diseases, including those associated with reduced amounts of functional adult hemoglobin (HbA), such as sickle cell disease and β-thalassemias

The invention relates to a long-acting oral medicine containing a hypoxia-inducible factor-proline hydroxylase inhibitor (HIF-PHI) and application of the long-acting oral medicine. Specifically, the present application discloses a long-acting oral pharmaceutical preparation containing a hypoxia-inducible factor-proline hydroxylase inhibitor (HIF-PHI) or a pharmaceutically acceptable salt thereof and one or more pharmaceutically acceptable carriers, adjuvants or excipients, the long-acting oral pharmaceutical preparation being suitable for administration at an administration interval of one week or more, therefore, the composition is suitable for long-term administration in treatment of chronic anemia.

There are provided polypeptides that include an Activin receptor type IIB (ActRIIB) ectodomain (ECD) variant. In some embodiments, a polypeptide of the disclosure includes an ActRIIB-ECD variant fused to an Fc domain moiety. The disclosure also provides pharmaceutical compositions and methods of using the polypeptides to treat diseases and conditions associated with TGFβ superfamily ligand signaling, such as metabolic disorders, diabetes, obesity, cardiometabolic disease, pulmonary hypertension, fibrosis, muscle weakness and atrophy, bone damage, and / or low red blood cell levels (such as anemia).

The present subject matter relates to recombinant lentiviral transfer plasmids for producing lentiviral vectors with increased vector titer and performance. These transfer plasmids are constructed so that a central polypurine tract (cPPT) is centered between the lentiviral LTRs and include a 6x stop codon sequence block downstream of the 3' LTR. When the transfer plasmids comprise a globin expression cassette, the lentiviral vectors produced therefrom are useful for treating hemoglobinopathies by gene therapy. Transduced cells, pharmaceutical compositions and methods of treating or ameliorating hemoglobinopathies with these lentiviral vectors are also provided. Additional transfer plasmids, cells, and compositions are provided that are useful for delivering any gene of interest to a cell, as well as plasmids for production of lentiviral vectors for treating other erythroid-specific diseases or disorders such as anemia and cancer.

Provided herein are methods for reducing the likelihood and / or severity of peripheral neuropathy, anemia, and / or neutropenia in an individual being treated with an antibody-drug conjugate (ADC). Also provided herein are methods for increasing dosing amount and / or frequency of an antibody-drug conjugate (ADC) in an individual. In some embodiments, the methods comprise administering to the individual an effective amount of an ADC that comprises an antibody conjugated to a drug moiety via a linker, wherein the antibody comprises a heavy chain comprising a human Fc region with reduced or eliminated effector function. Compositions, uses, and kits related thereto are also provided.

The invention relates to the technical field of microorganisms, and discloses Ackermann muciniphila, a combined microbial agent thereof and application of the Ackermann muciniphila in inflammatory enteritis. The invention provides Akkermansia muciniphila AIML-559, and the preservation number of the Akkermansia muciniphila AIML-559 is CGMCC (China General Microbiological Culture Collection Center) No. 34246. The invention relates to a combined microbial agent, which is characterized by comprising Ackermann muciniphila AIML-559 and Bifidobacterium adolescentis AIML-528 (the preservation number is CGMCC (China General Microbiological Culture Collection Center) No.34300). The combined microbial agent is prepared from the following raw materials: Ackermann muciniphila AIML-559 and Bifidobacterium adolescentis AIML-528 The ackermann mucophil combined microbial inoculum is suitable for treatment intervention of inflammatory bowel disease (IBD), and can relieve weight loss, hemolysis, anemia, diarrhea, intestinal bleeding and other symptoms of IBD mice.

The present disclosure provides methods of treating anemia in a patient in need thereof, comprising administering to the patient in need thereof an effective amount of salmeterol or a pharmaceutically acceptable salt thereof. Salmeterol or a pharmaceutically acceptable salt thereof may be administered conjointly with an erythropoiesis-stimulating agent, optionally wherein the anemia is refractory to the erythropoiesis-stimulating agent. Also provided are methods of promoting differentiation of an erythroid progenitor cell toward a mature red blood cell in a patient in need thereof, comprising administering an effective amount of salmeterol or a pharmaceutically acceptable salt thereof. The present disclosure further provides methods comprising administering salbutamol or a pharmaceutically acceptable salt thereof. Salmeterol, salbutamol, or a pharmaceutically acceptable salt thereof may be administered conjointly with other FDA-approved drugs such as luspatercept, lenalidomide, daprodustat, vadadustat, an erythropoiesis-stimulating agent (ESA), and / or a hypomethylating agent.

The invention relates to an application of a compound and a pharmaceutical composition thereof in preparation of a medicine for treating anemia of patients with non-myeloid malignant tumors subjected to chemotherapy, and the compound is N-[7-hydroxy-5-(2-phenethyl) [1, 2, 4] triazole [1, 5-a] pyridine-8-carbonyl] glycine.

The invention relates to the technical field of gene editing, in particular to a composition for treating alpha-thalassemia and application thereof. The composition is used for treating alpha-thalassemia and comprises a gene editing system. By adopting the composition disclosed by the invention, the repair editing of Hb-CS mutation can be realized, the expression of alpha / beta globin can be balanced, and the safety can be ensured.

The invention discloses application of colla corii asini bone-strengthening oral liquid to prevention and / or treatment of anemia, and belongs to the technical field of medicines, and the anemia comprises iron-deficiency anemia, renal anemia and myelosuppression anemia. The colla corii asini bone strengthening oral liquid can increase erythrocyte count, hemoglobin concentration, hematocrit, erythrocyte average hemoglobin amount, erythrocyte average hemoglobin concentration and / or erythrocyte average volume; the serum iron content is increased; a transporter divalent metal transporter and a membrane iron transporter are increased; the intestinal iron absorption is promoted. The composition specifically comprises urea nitrogen and creatinine; related indexes of renal anemia are improved; the immune index of renal anemia can be improved. The percentage of leukocytes and lymphocytes of anemia caused by myelosuppression can be recovered; the hemoglobin content is increased; the number of macrophage colony stimulating factors and / or bone marrow cells in serum is increased; the immunocompetence is improved; the proliferation of lymphoid B cells and macrophages is promoted.

Methods for treating patients with wAIHA comprising administering at least one compound chosen from (R)-2-[3-[4-amino-3-(2-fluoro-4-phenoxy-phenyl)pyrazolo[3,4-d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxetan-3-yl)piperazin-1-yl]pent-2-enenitrile (rilzabrutinib) and pharmaceutically acceptable salts thereof are disclosed.

The invention features ALK2 antibodies and antigen binding fragments thereof (e.g., ALK2 binding fragments). The invention also features pharmaceutical compositions and methods of using the ALK2 antibodies or antigen binding fragments thereof to treat bone disease or damage, low red blood cell levels (e.g., anemia or blood loss), heterotopic ossification (e.g., heterotopic ossification resulting from fibrodysplasia ossificans progressiva), Sjogren's syndrome (e.g., dry eye associated with Sjogren's syndrome), multiple osteochondroma, diffuse intrinsic pontine glioma, posterior capsule opacification, or cardiac hypertrophy and / or cardiac fibrosis.

The present disclosure describes novel hybrid soluble ActRIIB-ECD polypeptides which fully retain binding affinity for myostatin and activin A but demonstrate significantly reduced binding to BMPs, especially BMP-9. The novel compositions described herein can be used to prepare novel hybrid ActRIIB ligand trap proteins, which can be used for modulating the growth of muscle, bone, cartilage, fat, fibroblast, blood and neuronal tissue to counteract muscle wasting, bone loss, anemia, inflammation and fibrosis in a therapeutically meaningful manner. Because these novel next-generation myostatin / activin inhibitors are safer and more effective molecules than the currently available myostatin inhibitors, they are useful in a wide variety of clinical indications.

The invention features polypeptides that include an extracellular ActRIIA variant. In some embodiments, a polypeptide of the invention includes an extracellular ActRIIA variant fused to an Fc domain monomer or moiety. The invention also features pharmaceutical compositions and methods of using the polypeptides to treat diseases and conditions involving low red blood cell levels, e.g., anemia or blood loss; fibrosis; or pulmonary hypertension.

The invention provides a traditional Chinese medicine for treating children chronic aplastic anemia, and relates to the technical field of traditional Chinese medicines. The traditional Chinese medicine for treating children chronic aplastic anemia comprises the following components in parts by weight: 15-30 parts of astragalus membranaceus, 20-30 parts of golden cypress, 10-20 parts of angelica sinensis, 10-20 parts of prepared rehmannia root, 10-15 parts of codonopsis pilosula, 10-15 parts of bighead atractylodes rhizome, 10-15 parts of poria cocos, 10-15 parts of fructus lycii, 10-20 parts of dandelion, 10-20 parts of mint, 10-15 parts of glossy privet fruit, 10-15 parts of eclipta alba, 10-15 parts of fructus psoraleae, 10-15 parts of semen cuscutae, 10-15 parts of caulis spatholobi and 5-10 parts of colla corii asini. , 5-10 parts of tortoise-shell glue, and 5-10 parts of licorice. The radix astragali, the radix codonopsis, the rhizoma atractylodis macrocephalae, the poria cocos and the like have the effects of tonifying qi and spleen and promoting qi and blood biochemical sources; chinese angelica, prepared rehmannia root, donkey-hide gelatin, caulis spatholobi and the like can enrich and nourish blood The fructus lycii, the glossy privet fruit, the herba ecliptae, the fructus psoraleae, the semen cuscutae and the like have the effects of tonifying kidney and replenishing essence, regulating the immune function of the body and improving the hematopoietic microenvironment, so that the chronic aplastic anemia of children is effectively treated, and the levels of hemoglobin, leukocytes, platelets and other blood cells are improved.

Provided herein are crystalline salt and free base forms and amorphous forms of a compound having the following formula (I): (I) Compound 1. Also provided are pharmaceutical compositions comprising such crystalline and amorphous forms, processes for their manufacture and their use for the treatment of various conditions such as hemolytic anemia, sickle cell disease and MDS (very low risk MDS, low risk MDS, lower risk MDS and / or moderate risk MDS).

The invention discloses a traditional Chinese medicine composition for reinforcing qi, nourishing blood and strengthening body resistance after tumor chemoradiotherapy and a detection method thereof. The traditional Chinese medicine composition is prepared from astragalus membranaceus, codonopsis pilosula, angelica sinensis, poria cocos, cistanche, caulis spatholobi, radix paeoniae alba, glossy privet fruit, eclipta, hairyvein agrimony, medlar, Chinese yam, radix rehmanniae, radix ophiopogonis, deer-horn glue, tortoise-shell glue, pericarpium citri reticulatae and liquorice. The optimal traditional Chinese medicine composition and dosage are screened out through a large number of experimental screening and clinical medication experience, treatment is conducted on the basis of syndrome differentiation, compatibility is reasonable, the effects of reinforcing qi, nourishing blood and strengthening the body resistance are achieved together, immunity is improved, and the traditional Chinese medicine composition can be used for tumor chemoradiotherapy and postoperative conditioning and treatment of symptoms such as anemia, bone marrow suppression, thrombocytopenia and elderly weakness. Content determination is carried out through TLC identification and HPLC, and a scientific basis is provided for guaranteeing the clinical quality and effectiveness of reinforcing qi, nourishing blood and strengthening body resistance.

The invention provides an anti-anemia traditional Chinese medicine composition as well as a preparation method and application thereof. The anti-anemia traditional Chinese medicine composition is prepared from lithocarpus litseifolius, fructus gardeniae and rhizoma gastrodiae. The main active component dihydrochalcone (such as phlorizin, trilobatin, phloretin and the like) of lithocarpus litseifolius can improve iron metabolism by regulating a hepcidin-iron transporter axis; gardenoside in gardenia can target the liver to optimize iron distribution, and gastrodia elata polysaccharide can regulate immunity and improve the marrow microenvironment; the three forms a full-chain regulation and control network of intestinal tract iron absorption, liver iron utilization and bone marrow hematopoiesis microenvironment together, and the anti-anemia effect is enhanced in multiple dimensions.

The invention relates to a turnip juice containing probiotics that helps to resolve intestinal problems, supports the immune and cardiovascular systems, reduces cancer risk, protects the liver and kidneys, has antidiabetic effects, supports skin and hair health, reduces anemia, prevents osteoporosis, and possesses antimicrobial properties, as well as a method for producing this turnip juice.

The present invention relates to daprodustat or a pharmaceutically acceptable salt thereof for use in reducing fatigue in a specific population of non-dialysis subjects with anemia associated with chronic kidney disease, characterized by baseline hepcidin and hsCRP levels.

The invention relates to the technical field of biological medicines, and discloses an application of a compound wintercreeper mixture in preparation of a medicine for treating aplastic anemia. Two aplastic anemia mouse models are created by irradiating compound cyclophosphamide and chloramphenicol and busulfan compound cyclophosphamide; it is proved that the compound wintercreeper mixture can reduce the death rate of mice with aplastic anemia, improve blood indexes of the mice with aplastic anemia and obviously recover the bone marrow hematopoietic function of the mice.

The invention relates to application of anthraquinone compounds and pharmaceutically acceptable salts thereof in preparation of drugs for treating anemia, the anthraquinone compounds have a structure as shown in formula I. In the formula I, R1-R5 are independently selected from hydrogen, hydroxyl, unsubstituted or hydroxyl-substituted C1-C6 linear or branched alkyl, C1-C6 alkoxy, C1-C6 acyl or-(CH2) n1O (CH2) n2CH3, n1 is selected from 1, 2 or 3, and n2 is selected from 0, 1, 2 or 3. The anthraquinone compound provided by the invention can significantly up-regulate the transcriptional levels of HAMP, EPO and HBG1 genes, has a good treatment effect on thalassemia and beta-sickle anemia, has no significant influence on cell viability and mouse weight, and has medication safety.

Aspects of the present disclosure provide anti-TMPRSS6 antibodies and methods of using the same to promote hepcidin expression and treat iron overload related disorders such as hemopigmentation, sickle cell disease, thalassemia, hemolysis, Diamond-Blackfan anemia, myelodysplastic syndrome (MDS), and blood transfusion.

The present disclosure relates to 1-(2-(4-cyclopropyl-1H-1,2,3-triazol-1-yl)acetyl)-4- hydroxypyrrolidine-2-carboxamide derivatives and structurally related compounds of formula (I) as VHL inhibitors for the treatment of e.g. anemia (e.g. chronic anemia or anemia associated with chronic kidney disease, dialysis or cancer chemotherapy), ischemia, stroke or injury to the cardiovascular system during ischemia, or for enhancing wound healing, reducing scarring or enhancing angiogenesis or arteriogenesis.

The present invention discloses the application of sulforaphane in the preparation of a product for treating aplastic anemia, and belongs to the field of biotechnology. The present invention finds that sulforaphane (SPN) can not only reverse the disease degree of aplastic anemia, but also achieve the purpose of treating AA by inhibiting the occurrence of ferroptosis. Therefore, the use of sulforaphane in the preparation of a product for preventing and treating aplastic anemia must have a good application prospect, and the present invention provides a new solution for the treatment of aplastic anemia.