The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heterocyclic and heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heterocyclic and heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
A61K 31/4709 - Non-condensed quinolines containing further heterocyclic rings
A61K 31/5025 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
A61K 31/517 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or
2.
FORMULATIONS FOR STABILIZING DISSOLUTION OF MEDICAMENTS
The present disclosure provides a suspension granule formulation with good dissolution characteristics after suspension. Specifically, the present disclosure provides granules containing particles of a compound shown in Formula 1: or a pharmaceutically acceptable salt or solvate thereof, wherein 20% or less by weight of the particles comprising the granule formulation have a particle diameter of 500 μm or more.
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
05 - Pharmaceutical, veterinary and sanitary products
16 - Paper, cardboard and goods made from these materials
42 - Scientific, technological and industrial services, research and design
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Pharmaceutical preparations for the treatment of genetic disorders Printed materials, namely, labels and package inserts for pharmaceutical products Providing information about medical research Providing a website featuring information in the field of treatment of genetic disorders
Disclosed are crystalline forms of sepiapterin free base selected from polymorphs A, B, C, D, E, F, and G, and combinations thereof, as well as crystalline polymorphs of salts of sepiapterin. Also disclosed are pharmaceutical compositions containing one or more such polymorphs and methods for preparing such polymorphs. Sepiapterin is useful in the treatment of a number diseases associated with low cellular levels of BH4, for example, phenylketonuria.
C07D 475/04 - Heterocyclic compounds containing pteridine ring systems with an oxygen atom directly attached in position 4 with a nitrogen atom directly attached in position 2
7.
PHARMACEUTICAL COMPOSITIONS COMPRISING SEPIAPTERIN AND USES THEREOF
The present invention features pharmaceutical compositions including sepiapterin, or a pharmaceutically acceptable salt and/or co-crystal thereof, and methods for the treatment of tetrahydrobiopterin-related disorders (e.g., tetrahydrobiopterin deficiency or phenylketonuria) with such compositions.
The present invention features compositions including sepiapterin, or a pharmaceutically acceptable salt thereof, and methods for the treatment of BH4-related disorders. In some embodiments, these compositions and methods result in an increase in plasma exposure of BH4.
Provided herein are β-carboline compounds and their use to inhibit dihydroorotate dehydrogenase (DHODH):
Provided herein are β-carboline compounds and their use to inhibit dihydroorotate dehydrogenase (DHODH):
or a form thereof,
wherein R1, R2, R3, R4, Q1, Q2, Q3, Q4, R7, R9, and R11 are as defined herein.
The present invention relates to compounds that are useful as inhibitors of NOD-like receptor protein 3 (NLRP3) inflammasome pathway, processes for the preparation of said compounds, pharmaceutical compositions comprising said compounds, methods of using said compounds in the treatment of various diseases and disorders, and medicaments containing them, and their use in diseases and disorders mediated by NLRP3.
A61K 31/53 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine
The present disclosure provides a method for producing troloxamide by electrooxidation. In certain embodiments, the present disclosure allows the reaction to be carried out with reduced or no use of oxidants, providing a cleaner method of production compared to conventional methods.
09 - Scientific and electric apparatus and instruments
16 - Paper, cardboard and goods made from these materials
41 - Education, entertainment, sporting and cultural services
Goods & Services
Downloadable educational materials in the field of phenylketonuria and phenylketonuria patients, their caregivers, healthcare professionals and patient advocacy groups. Printed educational materials in the field of phenylketonuria and phenylketonuria patients, their caregivers, healthcare professionals and patient advocacy groups. Providing information regarding phenylketonuria to patients, their caregivers, healthcare professionals and patient advocacy groups.
16 - Paper, cardboard and goods made from these materials
41 - Education, entertainment, sporting and cultural services
Goods & Services
Printed educational materials in the field of phenylketonuria and phenylketonuria patients, their caregivers, healthcare professionals and patient advocacy groups. Providing information regarding phenylketonuria to patients, their caregivers, healthcare professionals and patient advocacy groups.
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biotechnology research; chemical engineering; providing
information on scientific and research studies in the field
of clinical medicine via a website; hosting a web site
featuring information on chemical engineering; research and
development in the pharmaceutical and biotechnology fields;
providing information on the subject of scientific research
in the field of biochemistry and biotechnology; research and
development of mrna drugs.
09 - Scientific and electric apparatus and instruments
41 - Education, entertainment, sporting and cultural services
Goods & Services
Downloadable electronic publications in the nature of books, manuals, booklets, pamphlets and brochures in the field of lifestyle topics for patients living with rare diseases, their caregivers and patient advocacy groups Arranging educational programs, namely, workshops, seminars, conferences, meetings in the nature of panel discussions, and programs that provide lifestyle-related information to patients living with rare diseases, their caregivers, and patient advocacy groups; Providing online non-downloadable videos in the field of lifestyle related information to patients living with rare diseases, their caregivers, and patient advocacy groups
09 - Scientific and electric apparatus and instruments
41 - Education, entertainment, sporting and cultural services
Goods & Services
Downloadable electronic publications in the nature of books, manuals, booklets, pamphlets and brochures in the field of lifestyle topics for patients living with Friedreich ataxia, their caregivers and patient advocacy groups Arranging educational programs, namely, workshops, seminars, conferences, meetings in the nature of panel discussions, and programs that provide lifestyle-related information to patients living with Friedreich ataxia, their caregivers, and patient advocacy groups; Providing online non-downloadable videos in the field of lifestyle related information to patients living with Friedreich ataxia, their caregivers, and patient advocacy groups
09 - Scientific and electric apparatus and instruments
41 - Education, entertainment, sporting and cultural services
Goods & Services
Downloadable electronic publications in the nature of books, manuals, booklets, pamphlets and brochures in the field of lifestyle topics for patients living with rare diseases, their caregivers and patient advocacy groups Arranging educational programs, namely, workshops, seminars, conferences, meetings in the nature of panel discussions, and programs that provide lifestyle-related information to patients living with rare diseases, their caregivers, and patient advocacy groups; Providing online non-downloadable videos in the field of lifestyle related information to patients living with rare diseases, their caregivers, and patient advocacy groups
09 - Scientific and electric apparatus and instruments
41 - Education, entertainment, sporting and cultural services
Goods & Services
Downloadable electronic publications in the nature of books, manuals, booklets, pamphlets and brochures in the field of lifestyle topics for patients living with Friedreich ataxia, their caregivers and patient advocacy groups Arranging educational programs, namely, workshops, seminars, conferences, meetings in the nature of panel discussions, and programs that provide lifestyle-related information to patients living with Friedreich ataxia, their caregivers, and patient advocacy groups; Providing online non-downloadable videos in the field of lifestyle related information to patients living with Friedreich ataxia, their caregivers, and patient advocacy groups
[0536] Provided herein are p-carboline compounds of Formula (I) and their use to inhibit dihydroorotate dehydrogenase (DHODH): [0537] or a form thereof, [0538] wherein R1, R2, R3, R4, and R5 are as defined herein.
A61K 31/437 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
The present disclosure relates to vatiquinone for use in treating a mitochondrial disease and other disorders characterized by high levels of oxidative stress and dysregulation of energy metabolism, wherein vatiquinone is administered with food.
A61K 31/122 - Ketones having the oxygen atom directly attached to a ring, e.g. quinones, vitamin K1, anthralin
A23L 33/10 - Modifying nutritive qualities of foodsDietetic productsPreparation or treatment thereof using additives
A61P 9/10 - Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
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
An alternatively spliced intronic sequence is disclosed, the splicing of which can be induced in the presence of a small molecule, e.g., Compound (I), as described herein.
42 - Scientific, technological and industrial services, research and design
Goods & Services
(1) Biotechnology research; chemical engineering; providing information on scientific and research studies in the field of clinical medicine via a website; hosting a web site featuring information on chemical engineering; research and development in the pharmaceutical and biotechnology fields; providing information on the subject of scientific research in the field of biochemistry and biotechnology; research and development of mrna drugs.
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biotechnology research; chemical engineering; providing information on scientific and research studies in the field of clinical medicine via a website; hosting a web site featuring information on chemical engineering; research and development in the pharmaceutical and biotechnology fields; providing information on the subject of scientific research in the field of biochemistry and biotechnology; research and development of mrna drugs.
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biotechnology research; Chemical engineering; Providing information on scientific and research studies in the field of clinical medicine via a website; Hosting a web site featuring information on chemical engineering; Research and development in the pharmaceutical and biotechnology fields; Providing information on the subject of scientific research in the field of biochemistry and biotechnology; Research and development of mRNA drugs.
The present description relates to methods of treating neurodegenerative diseases characterized by the accumulation of aberrant forms of the microtubule associated protein Tau (MAPT) using substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof.
A61K 31/4365 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system having sulfur as a ring hetero atom, e.g. ticlopidine
A61K 31/444 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. amrinone
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
The present description relates to methods of treating neurodegenerative diseases characterized by the accumulation of aberrant forms of the microtubule associated protein Tau (MAPT) using substituted pyrrolo[2,3-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof.
The present description relates to methods of treating neurodegenerative diseases characterized by the accumulation of aberrant forms of the microtubule associated protein Tau (MAPT) using substituted thieno[3,2-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof.
The present invention describes specific crystalline forms of 2-[3-(2,2,6,6- tetramethylpiperidin-4-yl)-3H-[1,2,3]triazolo[4,5-c]pyridazin-6-yl]-5-(2H-1,2,3-triazol-2- yl)phenol. The present invention further relates to methods for preparing said crystalline forms, pharmaceutical compositions comprising said crystalline forms, and methods of using said crystalline forms and pharmaceutical compositions comprising said crystalline forms to treat Huntington's Disease.
09 - Scientific and electric apparatus and instruments
41 - Education, entertainment, sporting and cultural services
Goods & Services
(1) Downloadable electronic publications in the nature of books, manuals, booklets, pamphlets and brochures in the field of lifestyle topics for patients living with phenylketonuria, their caregivers and patient advocacy groups. (1) Arranging educational programs, namely, workshops, seminars, conferences, meetings, and programs that provide lifestyle-related information to patients living with phenylketonuria, their caregivers, and patient advocacy groups; Providing online non-downloadable videos in the field of lifestyle related information to patients living with phenylketonuria, their caregivers, and patient advocacy groups.
09 - Scientific and electric apparatus and instruments
41 - Education, entertainment, sporting and cultural services
Goods & Services
(1) Downloadable electronic publications in the nature of books, manuals, booklets, pamphlets and brochures in the field of lifestyle topics for patients living with phenylketonuria, their caregivers and patient advocacy groups. (1) Arranging educational programs, namely, workshops, seminars, conferences, meetings, and programs that provide lifestyle-related information to patients living with phenylketonuria, their caregivers, and patient advocacy groups; Providing online non-downloadable videos in the field of lifestyle related information to patients living with phenylketonuria, their caregivers, and patient advocacy groups.
09 - Scientific and electric apparatus and instruments
41 - Education, entertainment, sporting and cultural services
Goods & Services
Downloadable electronic publications in the nature of books, manuals, booklets, pamphlets and brochures in the field of lifestyle topics for patients living with phenylketonuria, their caregivers and patient advocacy groups. Arranging educational programs, namely, workshops, seminars, conferences, meetings in the nature of panel discussions, and programs that provide lifestyle-related information to patients living with phenylketonuria, their caregivers, and patient advocacy groups; Providing online non-downloadable videos in the field of lifestyle related information to patients living with phenylketonuria, their caregivers, and patient advocacy groups
09 - Scientific and electric apparatus and instruments
41 - Education, entertainment, sporting and cultural services
Goods & Services
Downloadable electronic publications in the nature of books, manuals, booklets, pamphlets and brochures in the field of lifestyle topics for patients living with phenylketonuria, their caregivers and patient advocacy groups. Arranging educational programs, namely, workshops, seminars, conferences, meetings in the nature of panel discussions, and programs that provide lifestyle-related information to patients living with phenylketonuria, their caregivers, and patient advocacy groups; Providing online non-downloadable videos in the field of lifestyle related information to patients living with phenylketonuria, their caregivers, and patient advocacy groups
42.
METHODS FOR SYNTHESIS OF RACEMIC, SCALEMIC, AND CHIRAL ALPHA-TOCOTRIENOL QUINONE
The application discloses methods useful for the synthesis of racemic, scalemic, and chiral alpha-tocotrienol quinone, including synthetic intermediates and methods for making said synthetic intermediates.
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
The present invention relates to novel compounds of Formula (I): wherein each ring A, R1, R2, X, Y, and Z are as defined herein, which inhibit NOD-like receptor protein 3 (NLRP3) inflammasome activity. The invention further relates to the process for their preparation, pharmaceutical compositions and medicaments containing them, and their use in the treatment of diseases and disorders mediated by NLRP3.
Disclosed are quinoline and quinazoline compounds which modulate the activity of the gated ion channels. Compounds that modulate these gated ion channels are useful in the treatment of diseases and disorders related to pain, inflammation, the neurological system, the gastrointestinal system and genitourinary system. Preferred compounds include quinoline or quinazoline derivatives substituted at the 4-position via N(H), C(O) or O moieties.
A61P 25/14 - Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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
C07D 491/048 - Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or
The invention features methods of treating a SARS-CoV-2 infection in a subject, the method comprising administering to the subject an effective amount of sepiapterin or a pharmaceutically acceptable salt thereof.
The invention features methods of treating glioblastoma in a subject, the method comprising administering to the subject an effective amount of sepiapterin or a pharmaceutically acceptable salt thereof.
A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/495 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
The present disclosure provides an oxidative cleavage method of a Trolox amide. More particularly, the disclosure provides a method for producing a compound of formula I, the method including a step for contacting a compound of formula II with an oxidant in a solvent, and a step for contacting the solvent with a base to promote the reaction of the compound of formula II with the oxidant. In the formulae: R1, R2, R3, R4, R5, R6, R7, R8and R91-61-61-6 alkoxy, or -C(=O)NRARB; and RAand RB1-61-61-6 alkoxy.
C07C 231/12 - Preparation of carboxylic acid amides by reactions not involving the formation of carboxamide groups
C07C 235/78 - Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups and doubly-bound oxygen atoms bound to the same carbon skeleton with the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of an unsaturated carbon skeleton the carbon skeleton containing rings
C07D 311/66 - Benzo [b] pyrans, not hydrogenated in the carbocyclic ring other than with oxygen or sulfur atoms in position 2 or 4 with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached in position 2
51.
Pharmaceutical compositions comprising sepiapterin and uses thereof
The present invention features pharmaceutical compositions including sepiapterin, or a pharmaceutically acceptable salt and/or co-crystal thereof, and methods for the treatment of tetrahydrobiopterin-related disorders (e.g., tetrahydrobiopterin deficiency or phenylketonuria) with such compositions.
The present disclosure provides granules for suspension. Specifically, the present disclosure is a pharmaceutical composition comprising: a compound represented by formula 1 or a pharmaceutically acceptable salt or solvate thereof; a binder; and a dispersant. The binder includes a binder selected from the group consisting of a cellulose-based binder, a povidone-based binder, a vinyl alcohol-based binder, and a polysaccharide thickener-based binder. The dispersant is selected from the group consisting of an inorganic salt dispersant, an ether dispersant, a starch dispersant, a cellulose-based dispersant, and a fatty acid ester salt dispersant.
A61K 47/14 - Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
A61K 47/32 - Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers
A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
A61K 47/36 - PolysaccharidesDerivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
The application discloses methods useful for the synthesis of certain 2,3,5,6-alkyl-1,4-benzoquinone compounds, including 2,3,5-trimethyl-6-nonylcyclohexa-2,5-diene-1,4-dione, and synthetic intermediates for the production thereof.
C07C 39/19 - Compounds having at least one hydroxy or O-metal group bound to a carbon atom of a six-membered aromatic ring monocyclic with unsaturation outside the aromatic ring containing carbon-to-carbon double bonds but no carbon-to-carbon triple bonds
C07C 50/06 - Quinones with monocyclic quinoid structure with unsaturation outside the quinoid structure
C07D 319/08 - 1,3-DioxanesHydrogenated 1,3-dioxanes condensed with carbocyclic rings or ring systems
C07C 37/00 - Preparation of compounds having hydroxy or O-metal groups bound to a carbon atom of a six-membered aromatic ring
C07C 37/20 - Preparation of compounds having hydroxy or O-metal groups bound to a carbon atom of a six-membered aromatic ring by reactions increasing the number of carbon atoms using aldehydes or ketones
C07C 46/08 - Preparation of quinones by oxidation giving rise to quinoid structures of at least one hydroxy group on a six-membered aromatic ring with molecular oxygen
C07C 37/50 - Preparation of compounds having hydroxy or O-metal groups bound to a carbon atom of a six-membered aromatic ring by reactions decreasing the number of carbon atoms
C07C 37/055 - Preparation of compounds having hydroxy or O-metal groups bound to a carbon atom of a six-membered aromatic ring by replacing functional groups bound to a six-membered aromatic ring by hydroxy groups, e.g. by hydrolysis by substitution of a group bound to the ring by oxygen, e.g. ether group
The application discloses methods useful for the synthesis of certain 2, 3, 5, 6-alkyl-1, 4-benzoquinone compounds, including 2, 3, 5-trimethyl-6-nonylcyclohexa-2, 5-diene-1, 4-dione, and synthetic intermediates for the production thereof.
C07C 39/19 - Compounds having at least one hydroxy or O-metal group bound to a carbon atom of a six-membered aromatic ring monocyclic with unsaturation outside the aromatic ring containing carbon-to-carbon double bonds but no carbon-to-carbon triple bonds
C07C 50/06 - Quinones with monocyclic quinoid structure with unsaturation outside the quinoid structure
C07D 319/08 - 1,3-DioxanesHydrogenated 1,3-dioxanes condensed with carbocyclic rings or ring systems
C07C 37/00 - Preparation of compounds having hydroxy or O-metal groups bound to a carbon atom of a six-membered aromatic ring
C07C 37/20 - Preparation of compounds having hydroxy or O-metal groups bound to a carbon atom of a six-membered aromatic ring by reactions increasing the number of carbon atoms using aldehydes or ketones
C07C 46/08 - Preparation of quinones by oxidation giving rise to quinoid structures of at least one hydroxy group on a six-membered aromatic ring with molecular oxygen
Provided herein are compounds of Formula (I):
Provided herein are compounds of Formula (I):
and forms thereof, including compositions thereof and uses therewith for treating spinal muscular atrophy.
C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or
The present description relates to methods of treating spinocerebellar ataxia type 3 (SCA3) using substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof.
A method for modulating an amount or type of a mature RNA transcript, comprising contacting a compound of Formula (I) with a pre-mRNA transcript transcribed from a gene, wherein the gene comprises a DNA REMS sequence and the pre-mRNA transcript transcribed from the gene comprises a RNA REMS sequence.
C07D 419/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen, oxygen, and sulfur atoms as the only ring hetero atoms containing three or more hetero rings
A61K 31/4365 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system having sulfur as a ring hetero atom, e.g. ticlopidine
A61P 43/00 - Drugs for specific purposes, not provided for in groups
Disclosed are crystalline forms of sepiapterin free base selected from polymorphs A, B, C, D, E, F, and G, and combinations thereof, as well as crystalline polymorphs of salts of sepiapterin. Also disclosed are pharmaceutical compositions containing one or more such polymorphs and methods for preparing such polymorphs. Sepiapterin is useful in the treatment of a number diseases associated with low cellular levels of BH4, for example, phenylketonuria.
C07D 475/04 - Heterocyclic compounds containing pteridine ring systems with an oxygen atom directly attached in position 4 with a nitrogen atom directly attached in position 2
A61K 31/495 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
The present description relates to methods of treating spinocerebellar ataxia type 3 (SCA3) using substituted pyrrolo[2,3-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof.
The present description relates to methods of treating spinocerebellar ataxia type 3 (SCA3) using substituted thieno[3,2-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof.
09 - Scientific and electric apparatus and instruments
41 - Education, entertainment, sporting and cultural services
Goods & Services
(1) Downloadable electronic publications in the nature of books, manuals, booklets, pamphlets and brochures in the field of lifestyle topics for patients living with genetic diseases and disorders, their caregivers and patient advocacy groups. (1) Arranging educational programs, namely, workshops, seminars, conferences, meetings, and programs that provide lifestyle-related information to patients living with genetic diseases and disorders, their caregivers, and patient advocacy groups; Providing online non-downloadable videos in the field of lifestyle-related information to patients living with genetic diseases and disorders, their caregivers, and patient advocacy groups.
Disclosed herein is pharmaceutical compositions of Compound 1, and/or the hydroquinone form thereof, and methods useful for treating or suppressing a disease or disorder such as an α-synucleinpathy, a tauopathy, an autistic spectrum disorder, a pervasive developmental disorder, a liver disease, and liver damage in a subject using such pharmaceutical compositions.
An aspect of the present description relates to compounds useful for improving pre‑mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted heteroaryl compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating spinocerebellar ataxia type 3 (SCA3), also known as Machado–Joseph disease (MJD).
A61K 31/4365 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system having sulfur as a ring hetero atom, e.g. ticlopidine
A61K 31/517 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heterocyclic and heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
A61K 31/495 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
A61K 31/4162 - 1,2-Diazoles condensed with heterocyclic ring systems
The present description relates to a tablet formulation of 2-[3-(2,2,6,6-tetramethylpiperidin-4-yl)-3H-[1,2,3]triazolo[4,5-c]pyridazin-6-yl]-5-(2H-1,2,3-triazol-2-yl)phenol, a compound for use in treating Huntington's disease, and a method of making the same.
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
72.
Methods for increasing sepiapterin plasma exposure
The present invention features compositions including sepiapterin, or a pharmaceutically acceptable salt thereof, and methods for the treatment of BH4-related disorders. In some embodiments, these compositions and methods result in an increase in plasma, CSF, and/or brain exposure of sepiapterin.
Disclosed herein are polymorphic and amorphous forms of anhydrate, hydrate, and solvates of (R)-2-hydroxy-2-methyl-4-(2,4,5-trimethyl-3,6-dioxocyclohexa-1,4-dienyl)butanamide and methods of using such compositions for treating or suppressing oxidative stress disorders, including mitochondrial disorders, impaired energy processing disorders, neurodegenerative diseases and diseases of aging. Further disclosed are methods of making such polymorphic and amorphous forms.
A61K 31/122 - Ketones having the oxygen atom directly attached to a ring, e.g. quinones, vitamin K1, anthralin
C07C 215/30 - Compounds containing amino and hydroxy groups bound to the same carbon skeleton having hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being unsaturated and containing six-membered aromatic rings containing hydroxy groups and carbon atoms of six-membered aromatic rings bound to the same carbon atom of the carbon skeleton
C07C 215/08 - Compounds containing amino and hydroxy groups bound to the same carbon skeleton having hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being saturated and acyclic with only one hydroxy group and one amino group bound to the carbon skeleton
C07C 235/78 - Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups and doubly-bound oxygen atoms bound to the same carbon skeleton with the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of an unsaturated carbon skeleton the carbon skeleton containing rings
C07C 215/28 - Compounds containing amino and hydroxy groups bound to the same carbon skeleton having hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being unsaturated and containing six-membered aromatic rings
A61K 31/164 - Amides, e.g. hydroxamic acids of a carboxylic acid with an aminoalcohol, e.g. ceramides
C07C 231/12 - Preparation of carboxylic acid amides by reactions not involving the formation of carboxamide groups
C07B 57/00 - Separation of optically-active organic compounds
74.
2,3,5-TRIMETHYL-6-NONYLCYCLOHEXA-2,5-DIENE-1,4-DIONE FOR SUPPRESSING AND TREATING ALPHA-SYNUCLEINOPATHIES, TAUOPATHIES, AND OTHER DISORDERS
Disclosed herein are methods of treating or suppressing a disorder selected from the group consisting of α-synucleinopathies, tauopathies, ALS, traumatic brain injury, and ischemic-reperfusion related injuries.ury, comprising administering to a subject in need thereof a therapeutically effective amount of a compound of the formula: or the hydroquinone form thereof; or a solvate or hydrate thereof.
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
In particular, the present description relates to substituted monocyclic heteroaryl compounds of Formula (I) or Formula (II), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
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
C07D 401/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
C07D 409/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing three or more hetero rings
C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
C07D 417/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise a REMS, and the use of a compound described herein to modulate the functional protein produced from such altered endogenous genes.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
A61K 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
C07D 239/70 - Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings condensed with carbocyclic rings or ring systems
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
The disclosure describes the discovery of a dual targeting recombinant U7 RNA comprising at least two covalently linked USH2A-specific antisense sequences that act in synergy to disrupt the splicing of USH2A exon 13 and induce exon 13 skipping. Because the most prevalent pathogenic mutations causing USH2A retinitis pigmentosa reside in exon 13, for example, c.2299delG, removal of exon 13 from USH2A pre-mRNA not only deletes these mutations but also restores the open reading frame of USH2A, translation of USH2A exon13 translation and consequently the function of USH2A. Therefore, the AAV USH2A-specific recombinant U7 snRNA vector disclosed herein provides a gene therapy approach for the treatment of USH2A-associated retinal degeneration and hearing loss.
The present invention relates to compounds that are useful as inhibitors of NOD-like receptor protein 3 (NLRP3) inflammasome pathway, processes for the preparation of said compounds, pharmaceutical compositions comprising said compounds, methods of using said compounds in the treatment of various diseases and disorders, and medicaments containing them, and their use in diseases and disorders mediated by NLRP3.
A61K 31/407 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with heterocyclic ring systems, e.g. ketorolac, physostigmine
A61K 31/4162 - 1,2-Diazoles condensed with heterocyclic ring systems
A61K 31/53 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine
The present description relates to compounds of Formula (I), forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
The present description relates to compounds of Formula (I), forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
C07D 403/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
C07D 473/00 - Heterocyclic compounds containing purine ring systems
C07D 491/048 - Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
C07D 417/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
09 - Scientific and electric apparatus and instruments
41 - Education, entertainment, sporting and cultural services
Goods & Services
Downloadable electronic publications in the nature of Spanish language books, manuals, booklets, pamphlets and brochures featuring non-specific disease education materials and resources for patients living with genetic diseases and disorders, their caregivers and patient advocacy groups Arranging educational programs, namely, providing Spanish language workshops, seminars, conferences, meetings, and programs in the field of insurance, clinical trials, drug development, and patient advocacy to patients living with genetic diseases and disorders, their caregivers, and patient advocacy groups; Providing online non-downloadable Spanish language videos featuring insurance, clinical trials, drug development, and patient advocacy to patients living with genetic diseases and disorders, their caregivers, and patient advocacy groups
Provided herein are β-carboline compounds and their use to inhibit dihydroorotate dehydrogenase (DHODH): or a form thereof, wherein R1, R2, R3, R4, Q1, Q2, Q3, Q4, R7, R9, and R11 are as defined herein.
C07D 403/04 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings directly linked by a ring-member-to-ring- member bond
C07D 401/04 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings directly linked by a ring-member-to-ring- member bond
A61K 31/437 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
A61K 31/444 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. amrinone
A61P 43/00 - Drugs for specific purposes, not provided for in groups
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington’s disease.
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington’s disease.
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington’s disease.
In particular, the present description relates to substituted monocyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington’s disease.
C07D 403/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
C07D 401/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 417/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
C07D 417/04 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings directly linked by a ring-member-to-ring- member bond
C07D 401/04 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings directly linked by a ring-member-to-ring- member bond
C07D 409/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing three or more hetero rings
C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
C07D 405/12 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 403/10 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a carbon chain containing aromatic rings
C07D 403/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 417/10 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a carbon chain containing aromatic rings
C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
C07D 417/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
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 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61K 31/4709 - Non-condensed quinolines containing further heterocyclic rings
A61K 31/501 - PyridazinesHydrogenated pyridazines not condensed and containing further heterocyclic rings
A61K 31/517 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
One aspect described herein includes a method for treating coronavirus 2019 disease (COVID-19) caused by severe respiratory syndrome coronavirus 2 (SARS-CoV-2) by administering an effective amount of a DHODH inhibitor. Another aspect described herein includes a method of treating COVD-19 by administering 4-chlorophenyl (S)-6-chloro-1-(4-methoxyphenyl)-1,3,4,9-tetrahydro-2H-pyrido[3,4-b]indole-2-carboxylate (PTC299), or a pharmaceutically acceptable salt thereof.
A61K 31/437 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
A61K 31/675 - Phosphorus compounds having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for treating genetic diseases and disorders; pharmaceutical preparations for the treatment of genetic diseases and disorders
Provided herein are therapeutic and/or prophylactic compounds for mitochondrial or oxidative stress diseases such as cancer, amyotropic lateral sclerosis, Creutzfeldt-Jakob disease, Machado-Joseph disease, spinocerebellar ataxia, Huntington disease, Parkinson disease, Alzheimer disease, myocardial infarction, cerebral infarction, diseases related to aging, diabetes, alcoholic liver injury, chronic obstructive pulmonary disease, mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes (MELAS), and the like, wherein the compound is represented by formula (1), or reduced forms thereof, or pharmaceutically acceptable salts thereof.
A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
A61K 31/422 - Oxazoles not condensed and containing further heterocyclic rings
A61K 31/427 - Thiazoles not condensed and containing further heterocyclic rings
A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61K 31/4709 - Non-condensed quinolines containing further heterocyclic rings
A61K 31/538 - 1,4-Oxazines, e.g. morpholine ortho- or peri-condensed with carbocyclic ring systems
A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
A61P 25/00 - Drugs for disorders of the nervous system
C07D 235/02 - Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, condensed with other rings condensed with carbocyclic rings or ring systems
C07D 401/04 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings directly linked by a ring-member-to-ring- member bond
C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
C07D 403/04 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings directly linked by a ring-member-to-ring- member bond
C07D 403/10 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a carbon chain containing aromatic rings
C07D 403/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
C07D 405/04 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings directly linked by a ring-member-to-ring- member bond
C07D 407/04 - Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings directly linked by a ring-member-to-ring- member bond
C07D 413/04 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings directly linked by a ring-member-to-ring- member bond
C07D 413/10 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings linked by a carbon chain containing aromatic rings
C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
C07D 417/04 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings directly linked by a ring-member-to-ring- member bond
90.
METHODS FOR TREATING PARKINSON'S DISEASE WITH SEPIAPTERIN
A61K 31/197 - Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
A61K 9/00 - Medicinal preparations characterised by special physical form
The present description relates to methods of treating neurodegenerative diseases characterized by the accumulation of aberrant forms of the microtubule associated protein Tau (MAPT) using substituted pyrrolo[2,3-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof.
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 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
