A compound of formula (I) or pharmaceutically acceptable salts or hydrates thereof:
A compound of formula (I) or pharmaceutically acceptable salts or hydrates thereof:
wherein R is chosen from phenyl, a 6-membered heteroaryl group, cyclohexyl, a 5-membered heteroaryl group; a bicyclo [3.1.0] hexanyl group; a C2-C5 alkynyl group; and a cubanyl group; wherein R1 and R2, independently, are chosen from H; C1-C6 alkyl (optionally substituted with one or more halogens); C1-C6 alkyl-S(O)n—; CO2H (or C1-C6 alkyl esters thereof or C1-C6 alkyl amides thereof); halogen; C1-C6 alkoxy; CN; NO2; and NR7R8; wherein R7 and R8, independently, each represent H, C1-C6 alkyl, C1-C6 alkylcarbonyl, C1-C6 alkoxycarbonyl, arylsulphonyl, heteroarylsulphonyl, heterocyclosulphonyl, arylcarbonyl, heteroarylcarbonyl, heterocyclocarbonyl, or C1-C6 alkylsulphonyl, or R7, R8 and the nitrogen to which they are attached form a 5 or 6 membered heterocyclic ring (such as morpholine or piperidine); and wherein n represents 0-2.
C07D 473/06 - Heterocyclic compounds containing purine ring systems with oxygen, sulfur, or nitrogen atoms directly attached in positions 2 and 6 two oxygen atoms with radicals containing only hydrogen and carbon atoms, attached in position 1 or 3
A61K 8/49 - Cosmetics or similar toiletry preparations characterised by the composition containing organic compounds containing heterocyclic compounds
A61K 31/522 - Purines, e.g. adenine having oxo groups directly attached to the heterocyclic ring, e.g. hypoxanthine, guanine, acyclovir
A61P 43/00 - Drugs for specific purposes, not provided for in groups
C07C 273/18 - Preparation of urea or its derivatives, i.e. compounds containing any of the groups the nitrogen atoms not being part of nitro or nitroso groups of substituted ureas
C07D 239/545 - Two oxygen atoms as doubly bound oxygen atoms or as unsubstituted hydroxy radicals with other hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
2.
METHOD OF ALLELE SPECIFIC SILENCING FOR THE TREATMENT OF AUTOSOMAL DOMINANT CATECHOLAMINERGIC POLYMORPHIC VENTRICULAR TACHYCARDIA (CPVT)
The present invention provides a method for the treatment of autosomal dominant Catecholaminergic Polymorphic Ventricular Tachycardia associated with mutations in the cardiac ryanodine receptor type 2 (RYR2) gene, by the use of an AAV mediated RNA interference approach to induce allele specific silencing of mutant mRNA.
The invention concerns a compound of formula (I) or pharmaceutically acceptable salts or hydrates thereof: (I) wherein R represents a 6-membered aryl or heteroaryl group or a 5-membered heteroaryl group, optionally substituted with at least one halogen atom, for use as a medicament. The invention also concerns a pharmaceutical composition comprising, as active ingredient, at least one compound of formula (I), as above, or pharmaceutically acceptable salts or hydrates thereof, and at least one pharmaceutically acceptable excipient, and the pharmaceutical composition for use as a medicament. The invention also concerns such compound or pharmaceutical composition for use as HSP90 inhibitor, in selectively killing senescent cells, in delaying ageing in a subject and in the treatment and/ or prevention of a senescence -associated disease or disorder. The invention also concerns a method for preparing such compound. The invention also concerns the cosmetic use of the compounds of formula (I) or cosmetically acceptable salts or hydrates thereof, and of a cosmetic composition that comprises at least one such compound.
C07C 307/00 - Amides of sulfuric acids, i.e. compounds having singly-bound oxygen atoms of sulfate groups replaced by nitrogen atoms, not being part of nitro or nitroso groups
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
2516n21616162781-6161-616788 and the nitrogen to which they are attached form a 5 or • 6 membered heterocyclic ring (such as morpholine or piperidine); and • - n represents 0-2; for use as a medicament, in particular as HSP90 inhibitor, in selectively killing senescent cells, in delaying ageing in a subject and in the treatment and/or prevention of a senescence-associated disease or disorder.
A61P 25/00 - Drugs for disorders of the nervous system
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61P 37/00 - Drugs for immunological or allergic disorders
A61P 43/00 - Drugs for specific purposes, not provided for in groups
C07D 473/04 - Heterocyclic compounds containing purine ring systems with oxygen, sulfur, or nitrogen atoms directly attached in positions 2 and 6 two oxygen atoms
C07D 473/06 - Heterocyclic compounds containing purine ring systems with oxygen, sulfur, or nitrogen atoms directly attached in positions 2 and 6 two oxygen atoms with radicals containing only hydrogen and carbon atoms, attached in position 1 or 3
The disinfecting device (1) for disinfecting the air and/or surfaces of an environment to be treated according to the invention comprises an ozone generator (3) and an ultraviolet source (5). The device (1) diffuses in the surrounding environment the ozone produced by the ozone generator and irradiates the air of the environment to be treated with the ultraviolet rays produced by the ultraviolet source (5), and comprises A) a first treatment duct (7, 7 ', 7" ) configured for irradiating air coming from the environment to be treated with ultraviolet rays emitted by the ultraviolet source (5) and to send the irradiated air towards the environment to be treated; and B) a second treatment duct O) configured for mixing air coming from the environment to be treated with ozone produced by the ozone generator (3) and to send such a mixture towards the environment to be treated. The first (7, 7 ', 7") and second treatment ducts (9) are fluidically in parallel with each other so that the efficiency of one and of the other can be optimised.
A61L 2/24 - Apparatus using programmed or automatic operation
A61L 9/015 - Disinfection, sterilisation or deodorisation of air using gaseous or vaporous substances, e.g. ozone
F24F 8/22 - Treatment, e.g. purification, of air supplied to human living or working spaces otherwise than by heating, cooling, humidifying or drying by sterilisation using UV light
F24F 8/40 - Treatment, e.g. purification, of air supplied to human living or working spaces otherwise than by heating, cooling, humidifying or drying by ozonisation
6.
METHOD FOR DETERMINING A DISEASE PROGRESSION AND SURVIVAL PROGNOSIS FOR PATIENTS WITH AMYOTROPHIC LATERAL SCLEROSIS
THE MEDICAL RESEARCH, INFRASTRUCTURE AND HEALTH SERVICES FUND OF THE TEL-AVIV MEDICAL CENTER (Israel)
ISTITUTI CLINICI SCIENTIFICI MAUGERI (Italy)
HADASIT MEDICAL RESEARCH SERVICES AND DEVELOPMENT LTD. (Israel)
Inventor
Di Camillo, Barbara
Zandona', Alessandro
Daberdaku, Sebastian
Tavazzi, Erica
Chio', Adriano
Vasta, Rosario
Calvo, Andrea
Moglia, Cristina
Casale, Federico
D'Ovidio, Fabrizio
Mandrioli, Jessica
Lunetta, Christian
Drory, Vivian
Mora, Gabriele
Gotkine, Marc
Abstract
A method is described for determining a disease progression and survival prognosis, at a succession of prediction times, for patients suffering from amyotrophic lateral sclerosis (ALS). The method comprises a step of defining a set of variables associated with the onset and progression of amyotrophic lateral sclerosis, comprising a first group of variables associated with the onset of amyotrophic lateral sclerosis (comprising at least the variables "patient sex", "disease onset age", "disease onset site"), a second group of dynamic time variables (comprising at least the variable "time elapsed since disease onset"), a third group of dynamic functional variables (comprising at least one of the variables breathing, swallowing, communicating, walking/self-care or at least one variable of a functional progression and/or severity scale of amyotrophic lateral sclerosis), and further at least one variable associated with survival. The method further provides for encoding by means of a Dynamic Bayesian Network, using at least one trained algorithm, a plurality of probabilistic conditional dependence relationships, in which each relationship is a probabilistic conditional dependence relationship between two of the aforesaid variables. The aforesaid prediction times are defined so that each prediction time belongs to a respective time interval in which the conditional dependence relationships between the variables are stationary. The method further involves describing the Dynamic Bayesian Network, using at least one trained algorithm, by means of a corresponding graph, comprising said variables as nodes and comprising topological connections oriented between nodes corresponding to variables among which a probabilistic conditional dependence is identified. In the graph, given a node, the connections entering it show a conditional probability of the value assumed by the variable associated with such node, in a given prediction time, depending on the values assumed, in a prior prediction time, from the variables associated with the nodes from which such connections originate. The method further comprises the steps of entering, for each of the defined variables, data acquired at a given acquisition time relating to the situation of a specific patient; and calculating, by electronic processing and/or calculating means, on the basis of the Dynamic Bayesian Network and the graph, and starting from the aforesaid acquired data, the values of each of the defined variables, at one or more prediction times following the acquisition time. Finally, the method involves obtaining, in a given prediction time, disease progression prognosis results on the basis of the values of one or more of the variables of the third group calculated in such prediction time; and the survival prognosis results on the basis of the value of at least one variable associated with survival, calculated at such prediction time.
G16H 50/20 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems
G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment
7.
Method of allele specific silencing for the treatment of autosomal dominant Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
The present invention provides a method for the treatment of autosomal dominant Catecholaminergic Polymorphic Ventricular Tachycardia associated with mutations in the cardiac ryanodine receptor type 2 (RYR2) gene, by the use of an AAV mediated RNA interference approach to induce allele specific silencing of mutant mRNA.
Compositions and methods for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), particularly forms of the disease that are inherited in an autosomal dominant manner, by way of calsequestrin 2 (CASQ2) gene therapy can be used to treat CPVT caused by mutations in, for example, ryanodine receptor 2 and calmodulin, such as calmodulin 1 (CALM1) and CALM3. A variety of vectors are provided that can be used for the delivery of a CASQ2 transgene to a patient, such as a human patient suffering from autosomal dominant CPVT, including adeno-associated virus vectors, among others.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
ex vivoex vivo classifying breast microcalcifications (MCs) in a breast tissue sample, the method comprising the steps of: a) collecting Raman spectroscopy imaging data of a breast tissue sample containing at least one MC, the imaging data relating to the at least one MC; b) determining the composition of the at least one MC, based on reference spectra of at least one selected typical MC calcified components; c) producing an average Raman spectrum for each at least one MC by averaging at least one Type II MC component signal from the calcified components of the at least one MC; and d) carrying out a multivariate analysis with the average Raman spectrum for each at least one MC, based on a plurality of average Raman spectra averaging the same at least one Type II MC component signal from the calcified components of respective MCs, thus obtaining a classification of the at least one MC as benign MC or malignant MC.
Disclosed herein is a method for predicting a patient response to a sodium ion channel blocker such as mexiletine when the patient has LQT syndrome or an arrhythmia. The method generally comprises determining a plurality of parameters associated with sodium ion channels; generating a model for patient response by using a partial least squared (PLS) regression analysis on said plurality of parameters; and using the model to predict the patient response if the patient is administered a sodium ion channel blocker such as mexiletine.
The present invention concerns a method for the treatment of recessive Catecholaminergic Polymorphic Ventricular Tachycardia comprising delivering a gene into a cardiac cell.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
12.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DOMINANTLY-INHERITED CATECHOLAMINERGIC POLYMORPHIC VENTRICULAR TACHYCARDIA
The disclosure features compositions and methods for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), particularly forms of the disease that are inherited in an autosomal dominant manner, by way of calsequestrin 2 (CASQ2) gene therapy. The compositions and methods described herein can be used to treat CPVT caused by mutations in, for example, ryanodine receptor 2 and calmodulin, such as calmodulin 1 (CALM1) and CALM3. The disclosure provides a variety of vectors that can be used for the delivery of a CASQ2 transgene to a patient, such as a human patient suffering from autosomal dominant CPVT, including adeno-associated virus vectors, among others.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
The disclosure features compositions and methods for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), particularly forms of the disease that are inherited in an autosomal dominant manner, by way of calsequestrin 2 (CASQ2) gene therapy. The compositions and methods described herein can be used to treat CPVT caused by mutations in, for example, ryanodine receptor 2 and calmodulin, such as calmodulin 1 (CALM1) and CALM3. The disclosure provides a variety of vectors that can be used for the delivery of a CASQ2 transgene to a patient, such as a human patient suffering from autosomal dominant CPVT, including adeno-associated virus vectors, among others.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A01K 67/027 - New or modified breeds of vertebrates
14.
Method of gene transfer for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia (CPVT)
The present invention concerns a method for the treatment of recessive Catecholaminergic Polymorphic Ventricular Tachycardia comprising delivering a gene into a cardiac cell.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
15.
METHOD OF ALLELE SPECIFIC SILENCING FOR THE TREATMENT OF AUTOSOMAL DOMINANT CATECHOLAMINERGIC POLYMORPHIC VENTRICULAR TACHYCARDIA (CPVT)
The present invention provides a method for the treatment of autosomal dominant Catecholaminergic Polymorphic Ventricular Tachycardia associated with mutations in the cardiac ryanodine receptor type 2 (RYR2) gene, by the use of an AAV mediated RNA interference approach to induce allele specific silencing of mutant mRNA.
The present invention provides a method for the treatment of autosomal dominant Catecholaminergic Polymorphic Ventricular Tachycardia associated with mutations in the cardiac ryanodine receptor type 2 (RYR2) gene, by the use of an AAV mediated RNA interference approach to induce allele specific silencing of mutant mRNA.
09 - Scientific and electric apparatus and instruments
42 - Scientific, technological and industrial services, research and design
Goods & Services
Scientific and measuring equipment, airborne substances
sampler. Scientific research services and chemical analysis and
physical-chemical compounds sampled by the specimen sampler
in class 9 for the evaluation of air pollution levels.
18.
Method of gene transfer for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia (CPVT)
The present invention concerns a method for the treatment of recessive Catecholaminergic Polymorphic Ventricular Tachycardia comprising delivering a gene into a cardiac cell. The methods described herein relate to the use of viral vectors, such as adeno-associated virus (AAV) vectors, for the delivery of calsequestrin 2 (CASQ2) to cells, such as cardiomyocytes, so as to treat pathologies associated with mutations in this gene.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
19.
Glioprotectant peptide for use in the treatment of amyotrophic lateral sclerosis (ALS) and methods related thereto
The described invention relates to the use of the TAT-BH4 peptide for treating or preventing the progression of ALS. The methods include, postponing the appearance of symptoms and improving motor performance and survival in ALS. Methods are also provided, wherein the TAT-BH4 peptide is in a composition further comprising a pharmaceutically acceptable excipient.
A61P 25/00 - Drugs for disorders of the nervous system
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61K 38/16 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof
