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1. WO2019115854 - USE OF PHARMACOLOGICAL CHAPERONES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES

Publication Number WO/2019/115854
Publication Date 20.06.2019
International Application No. PCT/ES2018/070794
International Filing Date 10.12.2018
Chapter 2 Demand Filed 11.10.2019
IPC
A61K 31/351 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
335having oxygen as the only ring hetero atom, e.g. fungichromin
35having six-membered rings with one oxygen as the only ring hetero atom
351not condensed with another ring
A61P 3/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
CPC
A61K 31/351
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
335having oxygen as the only ring hetero atom, e.g. fungichromin
35having six-membered rings with one oxygen as the only ring hetero atom
351not condensed with another ring
A61K 31/70
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
A61P 3/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
C07D 309/12
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
309Heterocyclic compounds containing six-membered rings having one oxygen atom as the only ring hetero atom, not condensed with other rings
02having no double bonds between ring members or between ring members and non-ring members
08with hetero atoms or 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 to ring carbon atoms
10Oxygen atoms
12only hydrogen atoms and one oxygen atom directly attached to ring carbon atoms, e.g. tetrahydropyranyl ethers
Applicants
  • FUNDACIÓN BIOMÉDICA GALICIA SUR [ES]/[ES]
  • UNIVERSIDAD DE VIGO [ES]/[ES]
Inventors
  • ORTOLANO, Saida
  • BESADA PEREIRA, Pedro
  • TERAN MOLDES, Carmen
Agents
  • CARVAJAL Y URQUIJO, Isabel
Priority Data
P20173139611.12.2017ES
Publication Language Spanish (es)
Filing Language Spanish (ES)
Designated States
Title
(EN) USE OF PHARMACOLOGICAL CHAPERONES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES
(ES) USO DE CHAPERONAS FARMACOLÓGICAS PARA EL TRATAMIENTO DE ENFERMEDADES DE DEPÓSITO LISOSOMAL
(FR) UTILISATION DE CHAPERONS PHARMACOLOGIQUES POUR LE TRIATEMENT DE MALADIES LIÉES AU DÉPÔT LYSOSOMAL
Abstract
(EN) The present invention relates to the use of galactose analogues of formula (I) with the capacity to stabilize the structure of the α-GalA enzyme, for the treatment of lysosomal storage diseases and, in a preferred embodiment, for the treatment of Fabry disease. In addition, the present invention relates to pharmacological compositions comprising an effective amount of at least one of the galactose analogues described in the present document for the treatment of lysosomal storage diseases and, in a preferred embodiment, for the treatment of Fabry disease.
(ES) La invención se refiere al uso de análogos de la galactosa de la fórmula (I) que tienen la capacidad de estabilizar la estructura de la enzima α-GalA, para tratar enfermedades lisosomales de depósito y, en una realización preferida, para tratar la enfermedad de Fabry. Es también objeto de la presente invención las composiciones farmacológicas que comprenden una cantidad efectiva de al menos uno de los análogos de la galactosa descritos en el presente documento para tratar enfermedades lisosomales de depósito y, en una realización preferida, para tratar la enfermedad de Fabry.
(FR) La présente invention concerne l'utilisation d'analogues de la galactose de formule (I) qui ont la capacité de stabiliser la structure de l'enzyme α-GalA, pour traiter des maladies lysosomales de dépôt et, dans une réalisation préférée, pour traiter la maladie de Fabry. La présente invention se rapporte également à des compositions pharmacologiques qui comprennent une quantité efficace d'au moins un des analogues de la galactose délcrits dans le présent document pour traiter les maladies lysosomales de dépôt et, dans une réalisation préférée, pour traiter la maladie de Fabry.
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