272 results about "Severe anaemia" patented technology

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 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 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.

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 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.

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 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 invention provides a composition with an effect of improving iron-deficiency anemia as well as a preparation method and application of the composition, and belongs to the technical field of pharmaceutical compositions. The composition with the effect of improving iron-deficiency anemia, provided by the invention, is prepared from the following raw materials in parts by weight: 15 to 20 parts of radix astragali seu hedysari extract, 5 to 10 parts of fructus jujubae extract, 2 to 5 parts of radix paeoniae alba extract, 4 to 8 parts of radix angelicae sinensis extract and 8 to 16 parts of ethylenediaminetetraacetic acid ferric sodium salt. The defect of single effect in the prior art is broken through, the synergistic effect of the four effects of improving iron-deficiency anemia, helping sleep, improving acne and protecting gastric mucosa is achieved through scientific compatibility, the conditioning requirements of crowds with multiple diseases can be met without taking multiple products, the eating burden is reduced, meanwhile, the antagonistic effect among different product components is avoided, and the health-care product is suitable for being popularized and applied. The overall conditioning effect can be improved.

The invention relates to the use of ferroportin inhibitor compounds of the general formula (I) for treating transfusion-dependent β-thalassemia.

New applications of the HIF compound and the pharmaceutical composition thereof in preparation of drugs for treating diseases comprise applications in preparation of drugs for preventing or treating anemia induced by chemotherapy in non-myeloid malignant tumor subjects, altitude sickness and the like.

To provide dosing regimens for administering a HIF prolyl hydroxylase inhibitor in the treatment or prevention of anemia, such as anemia secondary to or associated with chronic kidney disease, or anemia associated with or resulting from chemotherapy.SOLUTION: A pharmaceutical composition in a form of a tablet or capsule comprising Compound 1 having the following structure, for use in a method of increasing hemoglobin levels in a patient in need thereof relative to a baseline hemoglobin level, wherein the patient has a hemoglobin level of ≤10.5 g / dL or ≥9.5 g / dL and ≤12 g / dL at a start of treatment, a serum hemoglobin level increases by at least about 0.1 to about 1.0 g / dL relative to the baseline hemoglobin level, and the pharmaceutical composition is used such that Compound 1 is administered to the patient once daily, for example at a dose of about 150 mg.SELECTED DRAWING: Figure 1a

The disclosure relates to a molecule that inhibits BAFF pathway, particularly an antibody or a binding fragment thereof, particularly an anti-BAFF-R antibody, e.g. ianalumab, for use in the treatment of autoimmune hemolytic anemia (AIHA), in particular, warm autoimmune hemolytic anemia (wAIHA).

The application discloses application of aza-3 in resisting erythroleukemia, wherein the aza-3 is a compound obtained by modifying a ring of aza-3 with 3alpha-O-(Boc-proline); the application proves that the aza-3 can obviously inhibit HEL and K562 cell proliferation, reduce splenomegaly, improve anemia indexes (RBC, HGB and HCT) and splenic and hepatic pathological infiltration, and has no obvious toxic side effects on main organs such as heart, lung and kidney through in-vitro MTT experiment and a Friend virus-induced erythroleukemia mouse model.

Provided herein are methods of treating anemia in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of an ALK2 inhibitor.

Provided herein is the use of particular dose levels or amounts of certain pyruvate kinase activators or pharmaceutically acceptable salts or compositions thereof, for treating anemia associated with low risk MDS, lower risk MDS and / or intermediate risk MDS (collectively, LRMDS) and other conditions.

The invention relates to the field of medical diagnosis, in particular to a thalassemia red blood cell morphology rapid analyzer based on a micro-fluidic chip, which comprises a micro-fluidic chip module, a shooting module, an image recognition module, a temperature control module and a display module, the temperature control module is used for controlling the temperature of the micro-fluidic chip module, and the shooting module is used for shooting the movement and deformation process of red blood cells in the micro-fluidic chip module. The image recognition module is used for carrying out image recognition on the shot image and analyzing the performance of the red blood cells from dynamic and static directions according to an image recognition result; and the display module is used for displaying a recognition result and an analysis result of the image recognition module. According to the scheme, the deformation capacity and the recovery capacity of the red blood cells are judged from the static direction and the dynamic direction, and the recognition accuracy of diseases such as thalassemia is improved.

The present invention relates to products and compositions and their uses. In particular, the invention relates to nucleic acid products that interfere with the expression of the TMPRSS6 gene or inhibit its expression and to therapeutic uses such as for the treatment of hemochromatosis, porphyria and hematological disorders such as β-thalassemia, sickle cell disease and iron overload from transfusions or myelodysplastic syndrome.

The invention relates to the technical field of hematopoietic reconstruction, in particular to application of costal cartilage derived stem cells and costal cartilage derived stem cells combined with pluripotent progenitor cells, hematopoietic stem cells or cyclosporine in preparation of a preparation for hematopoietic reconstruction. The costal cartilage derived stem cells (CD45-CD51 +) are derived from costal cartilage and have remarkable ecological niche recovery capability. When the cells are used alone or combined with hematopoietic stem cells and pluripotent progenitor cells, the hematopoietic function can be enhanced. The costal cartilage-derived stem cells are directionally differentiated into important supporting cells forming bone marrow ecological niche, secrete hematopoietic cell factors and protect hematopoietic stem / progenitor cells. In treatment of aplastic anemia, costal cartilage-derived stem cells and cyclosporin A have a synergistic effect, and immune-mediated bone marrow failure is improved through dual mechanisms. According to the technical scheme, the technical problem that the effect of treating bone marrow failure and promoting hematopoietic reconstruction by hematopoietic stem cell transplantation is not ideal is solved, and a new way is opened up for treating bone marrow failure and blood system diseases.

There remains a need for effective therapeutic agents for the treatment of TGFβ superfamily-associated disorders. [Solution] Polypeptides comprising an activin receptor type IIB (ActRIIB) ectodomain (ECD) variant are provided. In some embodiments, the polypeptides of the present disclosure comprise an ActRIIB-ECD variant fused to an Fc domain portion. The present disclosure also provides pharmaceutical compositions and methods using the polypeptides to treat diseases and conditions associated with TGF-β 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 (e.g., anemia).

In the field of gene therapy, a major hurdle is the design and identification of constructs and gene therapy vectors providing therapeutic effects while displaying satisfactory safety profiles. In the treatment of Diamond-Blackfan Anemia (DBA), therapies alleviating several crucial anemia symptoms, such as blood or bone marrow cellularity, hemoglobin levels, erythrocytes levels, or platelet levels, while showing satisfactory safety profiles remain a challenge. The present invention provides constructs encoding ribosomal protein genes involved in DBA, such as genes encoding RPS19, RPS17, RPS24, RPS10, RPL35a, RPL11, RPS26, and RPL5, vectors, methods, cells, and medical uses thereof, addressing these challenges and finding particular applications in the field of autologous cell therapy treatment of DBA. Further, the present invention provides a non-genotoxic conditioning protocol for preparing a subject prior to cell therapy treatment for DBA using construct of the present invention.

The invention provides a construction method of a mouse model for inducing acute inflammatory anemia. An existing inflammatory anemia model is too long in construction period and cannot simulate the clinical phenomenon of acute anemia, the mouse model provided by the invention can induce the number of red blood cells and the hemoglobin level to be remarkably reduced through single Poly (I: C) stimulation for 36 hours, an acute anemia time window is accurately simulated, and a good foundation is laid for mechanism research of acute inflammatory anemia.

It forms an object of the present invention a tetravalent antibody construct capable of selectively binding with high affinity to human EPOR, acting as a hEPOR agonist. It forms a further object said antibody for use in the treatment of kidney diseases and anemias.

The invention relates to the technical field of medicines, in particular to application of huperzine A in treatment of beta-thalassemia. It is found for the first time that the anti-Alzheimer disease drug huperzine A can be directly combined with gamma-globin to inhibit degradation of gamma-globin through a ubiquitin-proteasome pathway, so that the stability of gamma-globin and the fetal hemoglobin level are improved; the abnormal erythropoiesis and hemolysis phenotype of the beta-thalassemia are improved under the condition of not changing the mRNA expression. Animal experiments show that huperzine A obviously improves hematocrit and the number of red blood cells, reduces the size distribution width of the red blood cells and the proportion of reticulocytes, relieves oxidative stress, hemolysis, splenomegaly and iron overload, and has no obvious liver and kidney toxicity. The invention creatively provides a new strategy for improving HbF by regulating and controlling protein homeostasis instead of transcriptional up-regulation, and provides a new drug action mechanism and application direction for treatment of beta-thalassemia and other hemoglobin diseases.