67 results about "Related disorder" patented technology

Disclosed are methods of improving treatment adherence and compliance after discontinuation of incretin-based therapies, such as GLP-1 and / or GIP agonists, using pharmacotherapies comprising the disclosed therapeutic compounds and combinations. In some aspects, methods include treatment of psychological factors affecting other medical conditions (PFAOMC) following the administration of incretin-based therapies to treat obesity or hyperglycemia, and related disorders. In some aspects, disclosed pharmacotherapies for use in the methods include agents that modulate monoaminergic neurotransmission, administered alone or together, such as in disclosed combinations having advantageous synergistic effects.

The invention belongs to the technical field of biology, and particularly relates to an SLC44A1 gene and / or SLC44A5 gene knockout animal model as well as a construction method and application thereof. The construction method of the animal model comprises the following steps: deleting exon regions from the fourth exon region to the thirteenth exon region of the SLC44A1 gene and / or the second exon region of the SLC44A5 gene in mouse brain oligodendroglia cells in a high-efficiency manner by utilizing gene editing and a Cre-loxP recombinase system, so as to realize the knockout of a target gene. The animal model constructed by the invention can be used for researching the regulation mechanism of the choline transporters (SLC44A1 and SLC44A5) on myelin sheath formation, deeply knowing the biological functions of the choline transporters (SLC44A1 and SLC44A5), simulating the occurrence and development processes of human related diseases, revealing the pathogenesis of the related diseases, providing a new thought for prevention and treatment of brain myelin sheath dysplasia, and providing a new foundation for the prevention and treatment of brain myelin sheath dysplasia. And an effective drug or a treatment mode aiming at the disease is screened by utilizing a gene knockout animal model, so that a new choice is provided for clinical treatment of the disease.

The invention discloses a data-driven early-diabetic future diabetes-related disease progress risk prediction model and a construction method thereof, and the method comprises the steps: collecting clinical index data, including age, gender, BMI, WHR, HOMA-IR, HDL-C, TG, SBP, DBP, SCR and ALT, of early-diabetic patients in a training and verification queue; using an unsupervised soft clustering method combining dimension reduction based on UMAP, graph clustering and a Gaussian mixture model to identify the phenotypic heterogeneity of the prediabetes mellitus; obtaining the probability of the individual phenotype characteristics, evaluating the association between the probability and the development risk of the future diabetes related diseases in the early stage of diabetes, and constructing a development risk prediction model of the future diabetes related diseases in the early stage of urine diseases; and performing model optimization and robustness verification in the verification queue. According to the method, the heterogeneity of the prediabetes mellitus can be effectively identified, and accurate risk stratification and personalized prevention are realized.

The invention belongs to the technical field of biological medicines, and relates to application of LSR, LncRNA-LSR-AS1 or targeted siRNA in prevention and / or treatment of hyperlipidemia or related diseases thereof. The invention finds that the overexpression of the LSR gene shows a remarkable lipid-lowering effect in various hyperlipidemia models; the long-chain non-coding RNA (LncRNA-LSR-AS1) is specifically combined with a 3 'UTR (Untranslated Region) region of the LSR mRNA to negatively regulate the expression of the LSR gene; the targeting siRNA designed by aiming at the LncRNA-LSR-AS1 can be used for effectively inhibiting the expression of the LncRNA-LSR-AS1 and relieving the inhibiting effect of the LncRNA-LSR-AS1 on the LSR, so that the expression of the LSR is promoted. Effective intervention on hyperlipidemia can be achieved aiming at the target spots, the effects do not depend on an LDLR channel, and unique value is achieved for familial hypercholesterolemia with the poor PCSK9 inhibitor curative effect.

Described is small molecule 6-methoxy-7-(3-(pyrrolidin-1-yl)propoxy)-4-(tetrahydro-2H-pyran-4-yl)quinolin-2-amine (MS152) that inhibits methyltransferases G9a / EHMT2. This inhibitor can be used for the treatment of patients with G9a / EHMT2 related diseases such as Prader-Willi Syndrome and Alzheimer's Disease. Formula (I).

This invention relates to the field of pharmaceutical technology, and discovers that the natural products boldinine and norboldinine can be used as NTSR1 (Neurotensin receptor-1, NTSR1) receptor antagonists and their applications. The compounds are boldinine and norboldinine ((+)-Laurolitsine), which have NTSR1 receptor antagonistic activity and are NTSR1 receptor antagonists that can prevent and treat alcohol poisoning, addiction, schizophrenia, autism spectrum disorder, and mood disorders. Accordingly, this invention can provide novel NTSR1 receptor antagonist prospective compounds with clear targets for the above-mentioned related diseases.

The application discloses application of PTN in preparation of products for treating cognitive impairment caused by severe infection. The inhibitor of PTN provided by the application can inhibit chronic neuroinflammation, thereby preventing and inhibiting formation and development of late cognitive impairment caused by severe infection, and meanwhile, inhibition of expression or function of PTN can effectively block damage of infiltrating macrophages to microglia cells, thereby providing a new direction for treatment of related diseases.

A method for determination of an indicator representative of a change in the brain caused by a demyelinating disease, the method including, for each region of interest of the brain, determining a regional coefficient of one of the following diffusion coefficients: the radial diffusion, the axial diffusion, the mean diffusion, the anisotropy fraction, or a combination of several of these coefficients, the regional coefficients being determined from a diffusion MRI image; determining a number of changed regions, for which a condition relating to the value of the regional diffusion coefficient of each region is satisfied; and determining the indicator in accordance with the number of changed regions.

The present invention is characterized by PPT1 polypeptides and coding nucleic acid constructs. The use of the polypeptides and coding nucleic acid constructs includes producing PPT1 polypeptides, increasing PPT1 activity in subjects, and treating PPT1-related disorders such as CLN1 disease in subjects.

The present invention relates to the technical field of medicine, and in particular to a Polθ inhibitor, a preparation method therefor and a use thereof in medicine. The present invention provides a Polθ inhibitor represented by formula (Ia) or (Ib), a composition comprising same and a use thereof, wherein the compound can be used for treating or preventing Polθ-mediated diseases or disorders and related diseases or disorders.

The present invention recognizes that there is a need for the prophylaxis or treatment of Autism Related Disorders, which includes but is not limited to Autism Spectrum Disorder, Autism, Autistic Disorder, Asperger Syndrome, Rett Syndrome Fragile X syndrome, Phelan-McDermid Syndrome, Angelman Syndrome, Pitt Hopkins Syndrome, Prader-Willi Syndrome, or combinations thereof. A first aspect of the present invention generally relates to methods of prophylaxis or treatment of Autism Related Disorders using various pharmaceutical compositions. A second aspect of the present invention generally relates to pharmaceutical compositions used for the prophylaxis or treatment of Autism Related Disorders.

This invention discloses the application of MYDGF in the prevention and treatment of memory impairment and related diseases. This invention discloses that MYDGF shows significant differences in individuals with memory impairment. After administration of MYDGF, memory impairment can be prevented and memory impairment can be prevented and the death of newly generated neurons can be stopped.

Compositions and methods for treating, preventing, or reversing neuroinflammation-related disorders or autoimmune neurological disorders in a subject in need thereof are disclosed. The methods comprise administering a therapeutically effective amount of a composition comprising a myelin basic protein (MBP) peptide.

Provided herein are methods, compounds, and compositions for reducing expression of SCN2A in a subject. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate an SCN1A related disease or disorder (e.g., Dravet syndrome) in a subject in need.

The present invention relates to a novel compound represented by the following Chemical Formula 1 as a pentralin correcting agent, and a composition for preventing or treating deafness-goiter syndrome and diseases related thereto, comprising the compound as an active ingredient. [Chemical Formula 1] # imgabs0 #

Provided are methods of treating a subject with a SCN2A-related disorder, e.g., Developmental and Epileptic Encephalopathies (DEE), comprising administering to the subject an oligomeric compound. Also provided are methods of reducing frequency of seizures experienced by a subject with a SCN2A-related disorder, comprising administering to the subject an oligomeric compound.

The invention discloses mRNA (messenger Ribonucleic Acid) of an m6A methylation modified Ncam1 gene and an application of the mRNA. The invention discloses mRNA (messenger ribonucleic acid) of an m6A methylation modified Ncam1 gene. A modification site is selected from adenylic acid of at least one site of the 313th site, the 617th site, the 705th site, the 769th site, the 903rd site, the 1034th site, the 1202nd site or the 1430th site of the Ncam1 gene. The application comprises the application in up-regulation of Ncam1 protein expression and the application in up-regulation of Ncam1 protein expression. Application in inhibition of neuronal aging; application in preparation of a product for treating and / or improving Alzheimer's disease; the invention relates to application in preparation of drugs for regulating learning and memory ability decline related diseases. The mRNA provided by the invention regulates self expression through the change of m6A site methylation modification level and affects synaptic related genes and neuronal aging process, so that learning and memory processes are affected, and symptoms related to AD, aging and neurodegenerative diseases are improved.

Biomarkers that can support the diagnosis of inflammatory diseases and methods of their use. [Solution] A composition and method for detecting inflammasome components in a sample from a subject as a marker for inflammasome-related diseases or disorders such as multiple sclerosis, stroke, mild cognitive impairment, Alzheimer's disease, age-related macular degeneration, NASH, inflammatory aging, or traumatic brain injury. Methods for using such inflammasome markers to determine prognosis, prescribe treatment, and monitor response to treatment in subjects with inflammasome-related diseases or disorders such as multiple sclerosis, stroke, mild cognitive impairment, Alzheimer's disease, age-related macular degeneration, NASH, inflammatory aging, or traumatic brain injury are also described.

The application discloses application of a TPP2 mutant zebrafish in an ASD neurodevelopment-related disease model and belongs to the technical field of biological medicines. It is found for the first time that a TPP2 gene deletion type zebrafish has the following phenotypes: developmental retardation, developmental deformity, abnormal motor function, social dysfunction, stereotyped behavior, anxiety-like behavior and the like, has the core characteristics of ASD-related neurodevelopment-related diseases and can be used as an animal model for researching ASD neurodevelopment-related diseases, thereby providing an effective approach for diagnosing or treating ASD neurodevelopment-related diseases.

Use of H3K9me3 modulation for enhancing cognitive function and treating anxiety related disorders is presented. A composition is administered to the subject comprising a therapeutically effective amount of a SUV39H1 inhibitor comprising analogs of ETP69. The therapeutically effective amount is effective in treating cognitive dysfunction in aging and age-related disorders.

To provide treatment and detection of inherited neuropathies and associated disorders.SOLUTION: The present disclosure relates to methods of detecting and treating inherited neuropathy. In various aspects, the method comprises detecting the presence of a mutation in a sorbitol dehydrogenase (SORD) gene in a sample from a subject. In various embodiments, the SORD mutation is a DNA variant classified as pathogenic or likely pathogenic according to American College of Medical Genetics and Genomics (ACMG) criteria.SELECTED DRAWING: None

The invention relates to the technical field of medicine, in particular to a GLP-1 receptor agonist, a preparation method thereof and application of the GLP-1 receptor agonist in medicine. The invention provides a GLP-1 receptor agonist as shown in formula (F) and a composition and application thereof. The compound can be used for treating or preventing GLP-1 receptor mediated diseases or symptoms and related diseases or symptoms.

The present invention relates to a method for non-invasively assessing the risk of uniparental disomy and related diseases in a pregnant fetus. Specifically, the present invention provides a detection device for determining fetal uniparental disomy (UPD), characterized in that the device comprises: (a) a data input module; (b) a quality control module; (c) an analysis and calculation module; and (d) an output module. The analysis and calculation module includes copy number analysis and allele fraction analysis; and the allele fraction analysis includes detection of fetal uniparental disomy based on a hidden Markov model. As an auxiliary diagnostic method, the detection device of the present invention can detect fetal uniparental disomy and related disease risks with high sensitivity and specificity.

Disclosed are methods and compositions for the treatment of autism spectrum disorder, related disorders and symptoms of such disorders, comprising co-administration of an oxytocin peptide and magnesium ions. Co-administration of an oxytocin peptide and magnesium ions results in a synergistic or enhanced effect on reducing social and communication deficits in a patient suffering from an autism spectrum disorder.

The present invention relates to LPA-targeting siRNA and a conjugate, and specifically relates to LPA-targeting siRNA, a siRNA conjugate, a composition, and a pharmaceutical use thereof. The present invention also relates to a pharmaceutical composition, a cell or a kit comprising the siRNA, and a method for the siRNA and the siRNA conjugate for treating and / or preventing related disorders suffered by a subject.

Methods and systems for treating ALS and related motor neuron diseases using multi-site neuromodulation are disclosed.

The invention relates to a method for non-invasively assessing the risk of a pregnant fetus disophile and its related diseases. Specifically, the present invention provides a detection device for determining a fetal mononuclear diphile (UPD), characterized in that the device comprises: (a) a data input module; (b) a quality control module; (c) an analysis and calculation module; and (d) an output module. Wherein the analysis and calculation module comprises copy number analysis and allele score analysis; the allele fractional analysis comprises the step of detecting the monopathy of a fetus based on a hidden Markov model. As an auxiliary diagnosis method, the detection device disclosed by the invention can be used for detecting the risks of the fetal disophile and related diseases in a high-sensitivity and high-specificity manner.

The invention discloses application of Trolox in preparation of a medicine for treating neurodevelopment disorder related diseases, and belongs to the technical field of biological medicine. The invention firstly discovers that Trolox has new application of drugs for preventing and / or treating neurodevelopment disorder related diseases; furthermore, the Trolox restores the neural connectivity by repairing the densities of the damaged neuron dendritic spines of the brain hippocampus and / or striatum; abnormal dopaminergic signal channels are corrected, so that expression of key receptors and downstream molecules thereof is recovered to be normal, and therefore, diseases related to neurodevelopment disorders are prevented and / or treated; the neuroprotective effect of Trolox depends on the activation of dopamine D1 and D2 receptor pathways; in addition, the invention discloses the potential of Trolox in the aspect of targeted repair of the core pathology link of the neurodevelopmental disorder for the first time, and a clear candidate compound and an action target are provided for developing a novel medicine for treating the diseases.

To provide a magnesium-containing oxytocin preparation and a method of use. [Solution] Disclosed are methods and compositions for the treatment of autism spectrum disorder, related disorders, and symptoms of such disorders, comprising the simultaneous administration of oxytocin peptide and magnesium ions. The simultaneous administration of oxytocin peptide and magnesium ions results in a synergistic or enhanced effect on reducing social and communication deficits in patients with autism spectrum disorder. The present invention may address the need for oxytocin peptide formulations that can provide a more pronounced effect on the oxytocin treatment response in people with autism spectrum disorder and related disorders, which exhibit a wide range of variability.