130 results about "Thyreoidin" patented technology

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 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.

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.

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.

The invention belongs to the technical field of detection, and particularly discloses a filter paper dried blood spot thyroxine determination kit and a detection method thereof, and the filter paper dried blood spot thyroxine determination kit and the detection method provided by the invention are used for quantitative determination of neonatal thyroxine. Through the reconstitution fluid of the T4 antigen dry powder, the uniformity and the stability of manufacturing of the filter paper dried blood spot calibrator and the quality control product are realized; by optimizing antigen and antibody pairing, the detection sensitivity and precision of the reagent are effectively improved, and the linear range is widened; by optimizing a reaction carrier and a tracer agent process, the detection sensitivity of the reagent is improved, and the working range of a curve is widened; the elution release buffer solution is invented, so that elution, release and detection of TT4 in the filter paper dried blood spot are realized; and a full-automatic intelligent detection method is adopted, so that the detection speed, efficiency and precision are improved.

Provided are compounds having the following structure of Formula (I) or a pharmaceutically acceptable isomer, racemate, hydrate, solvate, isotope, or salt thereof, wherein A, X 1 , X 2 , Q, R 1 , R 2 , and n are as defined herein. Such compounds are useful as thyromimetic agents and have utility for treating diseases such as neurodegenerative diseases and fibrotic diseases. Also provided are pharmaceutical compositions containing such compounds, as well as methods of their use and preparation.

Assessment of transthyretin amyloid cardiomyopathy (ATTR-CM) can be performed by calculating a cardiac pyrophosphate activity (CPA) measurement from single-photon emission computed tomography (SPECT) images. SPECT images obtained using 99mTechnetium-pyrophosphate as a radiotracer can be analyzed to calculate CPA. Scan-specific thresholds for abnormal myocardial activity are identified based on left ventricular blood pool (LVBP) radiotracer counts, then radiotracer activity in regions of the myocardium with abnormal myocardial activity is determined. Finally, a CPA measurement can be calculated as a function of the mean radiotracer counts in such regions over the maximal LVBP radiotracer activity multiplied by the volume of involvement (e.g., the volume of abnormal activity). This CPA measurement can then be used as an assessment of ATTR-CM.

The invention discloses a novel serum metabolite composition based on dried blood spot detection and elaborates application of the serum metabolite composition as a marker in preparation of a congenital hypothyroidism diagnostic reagent or kit. The serum metabolite composition comprises thyroxine, 2-piperidone and phosphorylcholine PC (14: 0 / 20: 4). The serum metabolite composition can be used for screening and diagnosing patients with the congenital hypothyroidism, and has the characteristics of low detection cost, good repeatability and relatively high sensitivity and specificity.

Compositions and regimens useful in treating type I citrullenemia are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human Argininosuccinate Synthase 1 (ASS1).

The present subject matter is directed to methods of treating Allan-Herndon-Dudley syndrome comprising administering 3,5-diiodothyropropionic acid (DITPA) to a subject in need thereof, wherein the DITPA administration reduces triiodothyronine (“T3”) serum levels to normal, increases T3 brain levels to normal, and maintains normal serum levels of thyroxine (T4) and thyroid stimulating hormone (TSH). The subject may be a child or an adult.

Sulfophenyl and polyethylene glycol derivatives of the thyroid hormones L-thyroxine (T4) and triiodothyronine (T3), and methods of making same, are disclosed. Advantageously, the thyroid hormone derivatives are water soluble, have long-term stability, and don't stick to the surface of common container materials. The derivatives were prepared as calibrators, in non-serum-based solutions, for in vitro thyroid immunoassays to measure the concentration of test samples and were found to perform as well as thyroxine / serum-based solutions.

To provide an effective therapeutic method for TTR-associated diseases.SOLUTION: The present invention provides iRNA agents, for example, double-stranded iRNA agents, that target the transthyretin (TTR) gene, and methods of using such iRNA agents for treating or preventing TTR-associated diseases.SELECTED DRAWING: None

Immunotherapy for musculoskeletal disorders and conditions associated with transthyretin amyloidosis is provided.

The present invention relates to the field of molecular biology, and more particularly to nucleic acid aptamers, derivatives, and applications thereof for detecting TSHR protein. The present invention provides a nucleic acid aptamer for detecting TSHR protein, the nucleotide sequence of which is shown in SEQ NO. 1. The present invention provides a DNA aptamer YC6 with high affinity for TSHR protein, which has been validated at the molecular, cellular, and tissue levels. The present invention discovers that the DNA aptamer YC6 has the ability to recognize and target cells expressing thyrotropin receptors, providing new insights into the diagnosis and treatment of thyroid diseases and thyroid-related eye diseases.