74 results about "Amyloidosis" patented technology

A method of pre-screening a human subject for a clinical trial for treatment of tauopathy or an amyloidogenic disease. The method comprises obtaining a plasma sample from the subject and determining a concentration of p217+tau present in the plasma sample. The method further comprises indicating the subject for further screening for the clinical trial when the concentration of p217+tau present in the plasma sample is greater than or equal to a minimum threshold and less than or equal to a maximum threshold. The minimum threshold corresponds to an amount of p217+tau in plasma over which subjects present with mild cognitive impairment (MCI) and an increased accumulation of tau tangles in the brain as compared to a cognitively normal patient. The maximum threshold corresponds to an amount of p217+tau present in plasma over which subjects present with pathology of widespread accumulation of tau tangles in multiple regions of the brain.

The invention relates to a deep learning-based early-stage amyloidosis nephropathy identification and diagnosis method, belongs to the technical field of amyloidosis nephropathy identification, and solves the problem that early-stage amyloidosis cannot be accurately identified in the prior art. The method comprises the following steps: acquiring kidney pathological section images of different individuals to construct a training sample set; constructing a link model comprising a first region segmentation model, a second region segmentation model and a third region segmentation model; the first region segmentation model is used for segmenting a cortex region in the pathological image; the second region segmentation model is used for segmenting a glomerular region; the third region segmentation model is used for identifying amyloidosis of a glomerular region; training the link model based on the training sample set to obtain a trained link model; and inputting a to-be-identified kidney pathological section image into the trained link model to identify whether amyloidosis exists or not. And efficient and accurate early-stage amyloidosis identification is realized.

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.

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 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 discloses a myocardial amyloidosis quantitative analysis method based on SPECT imaging and related equipment, and relates to the field of wisdom medical device.The method comprises the steps that firstly, three-dimensional SPECT original data of N time phases of a target patient in the cardiac cycle are collected, a mu graph is generated based on low-dose CT for registration and attenuation correction, and a dynamic image is reconstructed; and then an MR structure sequence under synchronous electrocardio gating is obtained, and SPECT and MR space standardization and partial volume effect correction processing are completed. And unifying the cardiac cycle image form through affine and nonlinear transformation, and generating a high-quality enhanced SPECT image. Finally, radioactive counting information is extracted on the basis of three-dimensional segmentation, multiple myocardial quantitative indexes are calculated, comprehensive analysis of myocardial functions is achieved in combination with left ventricular functional parameters, and accurate image support is provided for non-invasive diagnosis and curative effect evaluation of myocardial amyloidosis.

The present invention relates to peptides that have preventive, ameliorative, or therapeutic effects against amyloidosis and / or macular degeneration, are safe for living organisms, and have few side effects including abnormal reactions, as well as pharmaceutical compositions and health functional foods containing the same.

Tetracycline derivatives and their use as a medicament. In particular, the tetracycline derivatives may be used in the treatment or the prevention of a disease, such as amyloidosis, pain, neurodegenerative diseases and neuroinflammatory diseases, in which the tetracycline derivatives or pharmaceutical compositions including these compounds are administered to a subject in need thereof.

A peptide-based vaccine for use in the treatment or prevention of amyloid-to-thyroid amyloidosis (ATTR) is provided.

The present disclosure relates in some aspects to methods and compositions for detecting amyloid-related diseases in an individual.

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

Transthyretin (TTR) is a soluble protein involved in thyroxine and retinol transport in the body. Under certain conditions, the TTR protein adopts a misfolded, misassembled, and / or aggregated TTR conformation, which can be toxic and lead to transthyretin-mediated amyloidosis (ATTR). Provided herein, inter alia, are compositions (e.g., pharmaceutical compositions) containing anti-TTR antibodies or antigenic fragments thereof, and related articles of manufacture. Further provided herein, inter alia, are methods for treating or preventing ATTR using the pharmaceutical compositions described herein.

The present disclosure relates to heterogeneous cell compositions derived from canine or feline uterine tissue and methods of producing and use thereof. In some aspects, the heterogeneous cell compositions comprise a mixture of mesenchymal progenitor cells and epithelial progenitor cells. In some aspects, the heterogeneous cell compositions are used as an autologous or allogeneic treatment for the treatment of diseases such as chronic kidney disease, atopic dermatitis, immune mediated arthritis, hepatitis, liver disease, inflammatory bowel disease, osteoarthritis, intravertebral disc disease, keratoconjunctivitis sicca (dry eye), pancreatitis, fibrosis, sclerosis, amyloidosis, immune mediated polyarthritis or wounds in canines and felines.

Aspects of the present invention disclose compounds that modulate the aggregation of amyloidogenic proteins or peptides. In some aspects, disclosed compounds modulate the aggregation of disease-associated proteins and natural β-amyloid peptides. In a preferred embodiment, the compounds can inhibit natural amyloid aggregation. Pharmaceutical compositions comprising the compounds of the embodiments, and diagnostic and treatment methods for diseases (e.g., amyloidogenic diseases) using the compounds, are also disclosed. In addition, there is provided an integrated bacterial platform for the discovery of rescuers of disease-associated protein misfolding.

Methods and materials for treating chronic cardiac disorders are provided herein. For example, methods and materials for using a dual guanylate cyclase A and B activator (e.g., a polypeptide having the sequence set forth in SEQ ID NO:1) to treat hypertrophic cardiomyopathy and other cardiac conditions, such as conditions associated with ventricular hypertrophy, atrial hypertrophy, ventricular remodeling, atrial remodeling, systolic and / or diastolic dysfunction, heart failure (e.g., heart failure with reduced ejection fraction, heart failure with mildly reduced ejection fraction, and heart failure with preserved ejection fraction), and other forms of chronic heart disease (e.g., Fabry disease, Noonan syndrome, Pompe disease, PRKAG2-related cardiomyopathy, Danon disease, Friedrich ataxia cardiomyopathy, amyloidosis, or desminopathy) are provided herein.

The compounds are used for the detection and imaging of amyloid plaques or both of proteins or peptides, for screening or testing the efficacy of inhibitors against amyloidosis and / or fibrillary growth of proteins or peptides, and / or for the detection of RNA and nucleolar imaging. The compounds are d 8 or d 10 Metal complexes or salts thereof. The metal complexes of said compounds can bind to amyloid proteins or peptides, plaques or both and / or RNA, nucleoli or both. This binding induces the accumulation and supramolecular self-assembly of the metal complexes, thereby causing changes in the photophysical properties of the metal complexes.

The present invention relates to (R)-7-(3,5-difluorophenoxy)-N-((1R,5S,8s)-3-(6- methoxypyridazin-4-yl)-3-azabicyclo[3.2.1]octan-8-yl)-6,7-dihydro-5H-pyrrolo[1,2- b][1,2,4]triazol-2-amine, or a pharmaceutically acceptable salt thereof, for use in the treatment of a disease associated with amyloidosis, and its method of treatment thereof.

Described herein are methods for treating transthyretin (TTR) amyloidosis with Compound 1 in a subject comprising certain mutations in the TTR protein (Compound 1). The methods include specific dosing regimens that have great efficacy in treating the subjects and that are well tolerated in subjects.

The present invention relates to a family of N-acylhydrazonic compounds structurally derived from 1-methyl-1H-imidazole-2-carboxaldehyde, or pharmaceutically acceptable salts thereof, and the use of said compounds to prevent and / or treat amyloid (such as Alzheimer's, Parkinson's and type 2 diabetes) and non-amyloid (such as cataracts) degenerative aggregopathies These compounds act as attenuators of the metal-protein interaction, preventing and / or decreasing protein oligomerization through competition with the target peptide or protein for the binding of physiological metal ions and, possibly, by modulating the protein-protein interaction itself. The invention also details four compounds specifically described as examples of N-acylhydrazones derived from 1-methyl-1H-imidazole-2-carboxaldehyde, namely: 1-methyl-1H-imidazole-2-carboxaldehyde isonicotinoyl hydrazone, 1-methyl-1H-imidazole-2-carboxaldehyde benzoyl hydrazone, 1-methyl-1H-imidazole-2-carboxaldehyde 2-furoyl hydrazone and 1-methyl-1H-imidazole-2-carboxaldehyde 2-thiophenyl hydrazone. The current application also comprises pharmaceutical compositions.

The present invention provides novel non-invasive methods for detecting misfolded transthyretin (TTR) proteins, and for diagnosing and monitoring amyloidosis and related disorders.

This disclosure pertains to the field of biomedicine, specifically relating to double-stranded oligonucleotides targeting the APP gene and their applications. Specifically, it provides double-stranded oligonucleotide agents or their salts, conjugates, or compositions for inhibiting amyloid precursor protein (APP) expression, wherein the double-stranded oligonucleotide agent comprises a sense strand and an antisense strand forming a double-stranded region; wherein the antisense strand sequence comprises at least 15 consecutive nucleotides of any of the sequences shown in SEQ ID NO:1-154 with a difference of no more than 3 nucleotides, and / or the sense strand sequence comprises at least 15 consecutive nucleotides of any of the sequences shown in SEQ ID NO:155-308 with a difference of no more than 3 nucleotides. The double-stranded oligonucleotide agent or its salt for inhibiting APP expression disclosed in this application can significantly inhibit APP expression and can be used for the prevention and / or treatment of diseases or conditions mediated by the APP gene and / or associated with protein amyloidosis.

The present disclosure relates generally to the field of neurology. In particular, the present disclosure relates to a method of detecting a neurodegenerative disease in a subject and a method of treatment thereof. The method comprises detecting the level of exosome-bound aggregation biomarkers in a sample obtained from the subject, wherein an increase in the level of exosome-bound aggregation biomarkers compared to a reference indicates that the subject has a neurodegenerative disease. Also described are methods for detecting a subject at risk of developing an amyloidosis or neurodegenerative disease, methods for detecting and treating an amyloidosis or neurodegenerative disease in a subject, and methods for determining the aggregation status of biomarkers in a sample.

Methods for the detection, monitoring, and treatment of cardiac fibrosis, progression of cardiac fibrosis, or heart failure in a subject comprising: (a) contacting a sample obtained from the subject with a binding agent that binds a region of cartilage intermediate layer protein 1 (CILP) that spans the cleavage site of the CILP precursor or a nucleotide encoding same. The cardiac fibrosis may be associated with one or more of: ischemia, congenital defect, familial fibrosis, infiltrative fibrosis, idiopathic fibrosis, amyloidosis, hemosiderosis, valvular disease, and other idiopathic cardiomyopathies.

Provided are: a transthyretin tetramer stabilizing agent; and a transthyretin amyloidosis preventing agent or progression suppressing agent.　The present invention relates to a transthyretin tetramer stabilizing agent containing a Glycyrrhiza glabra hydrophobic extract that includes Glycyrrhiza-glabra glabra polyphenol, and also to a transthyretin amyloidosis preventing agent or progression suppressing agent containing a Glycyrrhiza glabra hydrophobic extract that includes Glycyrrhiza-glabra glabra polyphenol.

The present invention provides compositions used for the treatment and / or prevention of protein aggregation disorders. [Solution] Proteopathy encompasses a wide range of ailments, including neurodegenerative diseases (e.g., polyglutamine diseases such as huntingtin in Alzheimer's disease, Parkinson's disease, and Huntington's disease, and prion diseases); amyloidosis of other non-neuronal proteins (especially I1-antitrypsin, immunoglobulin light and heavy chains, lactadherin, apolipoprotein, gelzolin, lysozyme, fibrinogen, atrial natriuretic factor, keratin, lactoferrin, and β-2 microglobulin, etc.); sickle cell disease; cataracts; cystic fibrosis; retinitis pigmentosa; and nephrogenic diabetes insipidus. Administration of sulfatase inhibitors is generally suitable for treating and / or preventing protein toxicity associated with proteopathy. Therefore, the present invention provides compositions comprising sulfatase inhibitors for the treatment of proteopathy.

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 (“Bispecific RBP4 / TTR ligand”) is a chemical entity that binds to both TTR and RBP4 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.

To provide a combination therapy with nirogacestat and BCMA-directed therapy and uses thereof.SOLUTION: The present disclosure provides methods of treating cancer or light chain amyloidosis in a subject in need thereof, comprising administering to the subject a combination therapy comprising an effective amount of Form A of nirogacestat dihydrobromide and a B-cell maturation antigen (BCMA)-directed therapy and the uses thereof. In one aspect, Form A of nirogacestat dihydrobromide is administered to the subject before, concurrently with, or after administering the BCMA-directed therapy.SELECTED DRAWING: None

The present invention relates to: a novel peptide which has a prophylactic, ameliorative or therapeutic effect on amyloidosis and / or macular degeneration, is safe to a living body, and has few side effects including abnormal reactions; a pharmaceutical composition comprising the same; and a health functional food.

Aspects of the present invention disclose compounds that modulate the aggregation of amyloidogenic proteins or peptides. In some aspects, disclosed compounds modulate the aggregation of disease-associated proteins and natural β-amyloid peptides. In a preferred embodiment, the compounds can inhibit natural amyloid aggregation. Pharmaceutical compositions comprising the compounds of the embodiments, and diagnostic and treatment methods for diseases (e.g., amyloidogenic diseases) using the compounds, are also disclosed. In addition, there is provided an integrated bacterial platform for the discovery of rescuers of disease-associated protein misfolding.

The invention belongs to the technical field of intelligent medical treatment, and discloses a dynamic risk assessment method and system for light-chain amyloidosis. A traditional staging system does not consider age factors and does not consider interaction between hematology and organ remission, so that obvious defects exist in the aspect of prognosis personalized risk prediction. According to the method, a 6-month risk scoring model and a 12-month risk scoring model are created for the first time, the age, hematological remission and heart and kidney organ remission depth of a patient are subjected to weighted integration through a multivariable Cox proportional risk model, and risk scores are generated and divided into corresponding risk grades. According to the method, the independent contribution and the interactive influence of each factor on the death risk are quantified, the method is closer to the complicated biological nature of the disease, continuous monitoring, dynamic evaluation and accurate prediction of prognosis are realized, the clinical use threshold is greatly reduced, the treatment decision can be directly linked, and an objective basis is provided for the treatment decision.