177 results about "Thyreoidin" patented technology

The invention relates to PRMT5 inhibitors of formula (I), a stereoisomer thereof, an atropisomer thereof, a pharmaceutically acceptable salt thereof, a pharmaceutically acceptable salt of the stereoisomer thereof, a pharmaceutically acceptable salt of the atropisomer thereof, or a deuterated molecule thereof, and pharmaceutical compositions thereof, as well as the use thereof.

An RNAi agent and composition for inhibiting the expression of transthyretin (TTR), which can be used for treating and / or preventing ATTR-related diseases comprising ATTR-PN (transthyretin amyloidosis polyneuropathy), ATTR-CM (transthyretin amyloidosis cardiomyopathy), senile systemic amyloidosis diseases, and leptomeningeal / central nervous system amyloidosis diseases.

Disclosed is a method for preventing, treating, or ameliorating alopecia areata, comprising administering a therapeutically effective amount of a thyromimetic compound or a pharmaceutically acceptable salt thereof to a subject in need thereof, wherein said thyromimetic compound is a compound of Formula I, wherein R1,R2,R3,R4,R5,R6,R7,R8,R9,G,T,and X are defined as herein.

Provided herein are anti-TTR antibody dosing regimens useful for the treatment of thyroxine transporter amyloid cardiomyopathy (ATTR-CM) in adult patients. In an embodiment, the patient is treated with an anti-TTR antibody comprising NI006 / ALXN2220.

The present invention relates to transthyretin (TTR) fusions useful in the dimerization and tetramerization of antibodies and antibody fragments, such as Fabs. The TTR fusions proteins described herein are particularly useful in increasing antibody avidity and in enhancing antigen clustering. Methods for treating diseases using the fusion proteins of the present invention are described herein.

The present invention concerns the use of aprotic polar solvents and an ionization stabilizing agent to prepare stable therapeutic formulations of levothyroxine by dissolving levothyroxine in an aprotic polar solvent system that can then be used to treat, prevent, and / or diagnose certain diseases and disorders in humans and veterinary animals by administration of the formulation thereto. In certain embodiments, the invention is directed to formulations comprising levothyroxine, and optionally one or more additional therapeutic agents, dissolved in an aprotic polar solvent system, such as a DMSO / water admixture comprising at least one ionization stabilizing excipient in a concentration sufficient to impart physical and chemical stability to the therapeutic agent. The invention also provides methods of use of such formulations in treating, preventing, and / or diagnosing diseases and disorders, and methods of manufacturing such formulations.

The invention provides application of a biomarker combination for early screening and diagnosis of transthyretin cardiac amyloidosis (ATTR-CA) high-risk groups in preparation of a product for early screening and diagnosis of the ATTR-CA high-risk groups. In general, one or more biomarkers, such as total prealbumin (total PA), transthyretin (TTR) tetramer, misfolded protein, protein misfolding rate, and soluble growth stimulus expression gene 2 protein (sST2). In ATTR-CA high-risk groups and non-high-risk groups, the biomarkers show significant difference, which prompts that the biomarkers are related to ATTR-CA. When a plurality of biomarkers are combined for use, the biomarkers have relatively high sensitivity and specificity in early screening and diagnosis of ATTR-CA high-risk groups, have relatively high diagnosis efficiency, and provide a new thought and strategy for early diagnosis and treatment of ATTR-CA.

Compounds are provided having the structure of Formula (I) or a pharmaceutically acceptable isomer, racemate, hydrate, solvate, isotope, or salt thereof, wherein R1, R2, X1, X2, Y1, and Y2 are as defined herein. Such compounds function as thyromimetics and have utility for treating diseases such as neurodegenerative disorders and fibrotic diseases. Pharmaceutical compositions containing such compounds are also provided, as are methods of their use and preparation.

A stable sustained release injectable formulation based on poly(D,L-lactide-co-glycolide) microparticles comprising levothyroxine is described. The formulation has a theoretical levothyroxine loading of at least 1.5% w / w. A process for preparing the microparticles is also described. The formulation may be used to control hypothyroidism in adults, congenital hypothyroidism in infants, and acquired hypothyroidism in children.

The invention relates to methods of treating subjects that would benefit from reduction in expression of transthyretin (TTR), such as subjects having a TTR-associated disease, disorder, or condition, e.g., transthyretin-mediated amyloidosis, or Stargardt disease, using double-stranded ribonucleic acid (dsRNA) compositions targeting the TTR gene. The invention also provides methods for preventing at least one symptom in a subject having a TTR-associated disease, disorder, or condition, e.g., transthyretin-mediated amyloidosis, or Stargardt disease.

Described herein are methods for treating transthyretin (TTR) amyloidosis in a subject. The methods include specific dosing regimens that have great efficacy in treating the subjects and that are well tolerated in subjects.

The invention provides a pharmaceutical composition. The pharmaceutical composition is composed of a thyroxine receptor beta agonist, folic acid substances and pharmaceutically acceptable auxiliary materials. The invention also relates to application of the composition in preparation of drugs for treating chronic fatty liver diseases. The pharmaceutical composition has the advantages that the pharmaceutical composition can be used for treating the fatty liver accompanied by hypothyroidism, and adverse reactions generated by independently taking a thyroid receptor beta agonist can be reduced.

Based on co-drugs representing two different chemical entities and the co- and sequential administration of the two chemical entities, novel therapies for macular degeneration and TTR amyloidosis are provided. The first component (“selective TTR ligand”) is a chemical entity that binds to TTR in the RBP4 (retinol-binding protein 4)-TTR (transthyretin) complex, which participates in the delivery of retinol to the retina. This component reduces retinol transport from circulation to the retina and provides stabilization of the TTR tetramer. The second component (“C20-D3-visual chromophore-generating compound”) is a C20-D3 modified retinoid or carotenoid that, when metabolized in mammals, ultimately produces a C20-D3 visual chromophore, which is presented in the retina as C20-D3-9-cis-retinal or C20-D3-11-cis-retinal. Deuteration at C20 reduces the formation of lipofuscin biretinol, but other functions (such as providing a precursor for the synthesis of the visual chromophore 11-cis-retinaldehyde in vivo) are not reduced.

Provided herein are compounds, preferably thyroid hormone receptor beta, THRbeta, agonist compounds, compositions thereof, and methods of making the same, and methods of agonizing THRbeta, and methods for treating conditions mediated by THRbeta.

The present invention relates to thyroxine beta receptor selective agonist compounds, pharmaceutical compositions and uses thereof. Specifically, the invention relates to a compound as shown in a formula (I). The compound provided by the invention has excellent drug effect and / or specificity, and can be used for treating metabolism-related diseases.

The invention relates to a method of treating ocular amyloidosis by reducing TTR expression in a subject by administering a double-stranded ribonucleic acid (dsRNA) that targets a TTR gene to the retinal pigment epithelium of the subject.

A treated human umbilical cord composition for use in the treatment of Perony's disease by in vivo injection in a subject in need thereof using an effective amount of the composition. The composition comprises a water-based human umbilical cord filtrate, wherein endogenous hyaluronic acid (HA) and / or hyaluronic acid, fibronectin, insulin growth factor binding protein-1 (IGFBP-1), sulfated glycosaminoglycan (sGAG), exosomes, interleukin-1 receptor antagonist (IL-1ra), hepatocyte growth factor (HGF), transthyretin, metalloproteinase tissue inhibitor 1 (TIMP-1), and / or a pharmaceutically acceptable salt thereof in an amount effective to reduce the size of Pteroid disease plaques. The present invention relates to an aggregation glycan, or a combination thereof.

A modified-release tablet dosage form containing tolcapone is disclosed. The tablet dosage form provides a pulsatile, pH-dependent release profile of tolcapone to both the gastric cavity and the small intestine. Methods for treating or preventing a disease selected from transthyretin amyloidosis (ATTR), Parkinson's disease, and obsessive-compulsive disorder using the dosage form are provided.

The invention relates to a conjugate for transferring an effector molecule from outside a cell into said cell, the conjugate comprising at least one effector molecule to be transferred into the cell, at least one saponin of the mono-desmosidic triterpene glycoside type or the bi-desmosidic triterpene glycoside type, and at least one single-domain antibody (sdAb), covalently bound to each other, wherein the sdAb is capable of binding to a cell-surface molecule of said cell. The invention also relates to a pharmaceutical composition comprising the conjugate of the invention. Furthermore, the invention relates to a pharmaceutical composition of the invention, for use as a medicament. In addition, the invention relates to a pharmaceutical composition of the invention, for use in the treatment or the prophylaxis of any one or more of: a cancer, an auto-immune disease such as rheumatoid arthritis, an enzyme deficiency, a disease related to an enzyme deficiency, a gene defect, a disease relating to a gene defect, an infection such as a viral infection, hypercholesterolemia, primary hyperoxaluria, haemophilia A, haemophilia B, alpha-1 antitrypsin related liver disease, acute hepatic porphyria, an amyloidosis and transthyretin-mediated amyloidosis. The invention also relates to an in vitro or ex vivo method for transferring the conjugate from outside a cell to inside said cell or for transferring the effector molecule comprised by the conjugate of the invention from outside a cell to inside said cell, preferably to the cytosol of said cell.

The application discloses a construction method and application of an osteoarthritis cartilage organoid, and the method comprises the following steps: 1) mesenchymal stem cells are inoculated on a round bottom culture plate by a Micromass method, and are cultured in a chondrogenic induction culture medium containing dexamethasone, ascorbic acid and TGF-beta 3 for 10-18 days to form a transparent cartilage organoid; and 2) the transparent cartilage organoid is placed in an osteoarthritis induction culture medium containing dexamethasone, L-thyroxine, inflammatory factors (IL-1beta, IL-6 and TNF-alpha) and beta-glycerophosphate sodium for continuous culture for 5-10 days to obtain an osteoarthritis cartilage organoid; the model simultaneously presents multi-dimensional pathological characteristics such as matrix degeneration, hypertrophy, inflammation activation and vascularization trend, and can be used for osteoarthritis pathogenesis research, drug screening and cell treatment effect evaluation; the construction method is controllable and has good repeatability, and provides a reliable three-dimensional in-vitro model for osteoarthritis research.

The invention discloses an on-line separation and valuing method for free thyroxine in serum. The method comprises the following steps: (1) setting a two-dimensional liquid chromatogram; (2) on-line separation and removal of macromolecular interferents in the serum; (3) retaining free thyroid hormone; (4) on-line separation of free thyroid hormone; (5) on-line valuing by isotope dilution mass spectrometry; and (6) cleaning the two-dimensional liquid chromatography system. The method provided by the invention realizes on-line separation of free thyroid hormone in serum and absolute constant value. A first-dimensional chromatographic mobile phase is a mild ammonium salt buffer solution and does not contain an organic solvent, so that thyroid hormones combined with protein in serum can be kept in a combined state, and a target object entering mass spectrometry is free thyroid hormones; and the valuing result can be traced to the international unit system through the national certified standard substance. Compared with traditional serum off-line treatment modes such as solid-phase extraction and equilibrium dialysis, the method has the characteristics of high efficiency, reliability and easiness in operation.

The present invention relates to the field of antibodies, antigen-binding fragments and immunodiagnosis. Specifically, the present invention relates to recombinant antibodies or antigen-binding fragments for binding thyroid hormone T4 (thyroxine) and halogenated bisphenol A. Furthermore, the present invention relates to isolated nucleic acid molecules comprising nucleotide sequences encoding the recombinant antibodies or antigen-binding fragments of the present invention, as well as expression vectors and host cells comprising the nucleic acid molecules of the present invention. In addition, the present invention relates to methods for producing recombinant antibodies or antigen-binding fragments for binding T4 thyroid hormone and halogenated bisphenol A, test kits and immunoassays comprising the recombinant antibodies or antigen-binding fragments of the present invention, and methods for determining the levels of T4 thyroid hormone and / or halogenated bisphenol A in a sample of a subject. Furthermore, the present invention relates to methods for processing samples, such as immunoaffinity-based sample preparation methods for enriching halogenated bisphenol A from a sample.

A pharmaceutical composition for administering directly to the pulmonary tract of a subject includes a salt of triiodothyronine and a pharmaceutically acceptable buffer, adjusted to a pH of 5.5-8.5. The composition can be administered prophylactically or therapeutically to a subject to treat lung inflammation or pulmonary edema.

Disclosed is a method for diagnosing a cancer health state, or a change in cancer health state in a patient, or for diagnosing a risk of the change or presence of a cancer in a patient, comprising determining, in a plasma sample from said patient, one or more biomarker values that correspond to transthyretin-containing complex structures, and assigning the patient as having or not having cancer, or having or not having a change in cancer health state, or having or not having a risk of cancer based on said biomarker values, wherein said cancer is preferably lung cancer (LC).

The present disclosure relates to the field of clinical detection technology, in particular, to the application of triiodothyronine autoantibody as a marker of thyroid dysfunction during cancer treatment. The present disclosure can help clinicians to judge the real thyroid function status of patients and improve the accuracy of diagnosis.

The disclosure relates to RNAi constructs, such as siRNA, for reducing expression of the TTR gene. Methods of using such RNAi constructs to treat or prevent cardiac disease, such as transthyretin-associated cardiomyopathy (ATTR-CM), are also described.

Provided herein are compounds of formula (I): or a stereoisomer or tautomer thereof, or a pharmaceutically acceptable salt of any of the foregoing, wherein T, L, LB, E, and D1 are as defined elsewhere herein. Also provided are methods of preparing compound of formula (I).

The present invention addresses the problem of providing a means for making it possible to detect transthyroxine (TTR) tetramers in a sample. The problem is solved by a step for forming a complex containing a TTR tetramer in a sample, a compound capable of binding to a thyroxine binding site of the TTR tetramer, an antibody capable of binding to a TTR monomer, and a labeling substance, and a step for measuring a signal generated by the labeling substance contained in the complex.

An amino dithioperacid thioester compound, a preparation method therefor, and use thereof. The structural formula of compound is as shown in formula I: wherein m=1-11, X is a nitrogen-containing aliphatic heterocyclic ring, and a nitrogen atom in the aliphatic heterocyclic ring is connected to a carbon atom of a thiocarbonyl group. The compounds disclosed by the invention are found to be capable of relieving muscular atrophy and lipolysis caused by cancer cachexia through in-vivo and in-vitro experiments. The compounds are also capable of obviously relieving weight and food intake reduction caused by cancer cachexia in animal experiments, so that the carbamo(dithioperoxo)thioates compounds have the effect on resisting cancer cachexia, can be applied to the treatment of cancer cachexia and related diseases, and become one kind of ideal cancer cachexia treatment medicament.