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1. WO2020113340 - USE OF SODIUM 2-(3-PENTYLPHENYL)ACETATE IN THE TREATMENT OF ALSTRÖM SYNDROME

Publication Number WO/2020/113340
Publication Date 11.06.2020
International Application No. PCT/CA2019/051753
International Filing Date 05.12.2019
IPC
A61K 31/192 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
19Carboxylic acids, e.g. valproic acid
192having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid
A61K 9/20 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
20Pills, lozenges or tablets
A61K 9/48 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
A61P 3/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
A61P 3/08 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
08for glucose homeostasis
C07C 57/30 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
CACYCLIC OR CARBOCYCLIC COMPOUNDS
57Unsaturated compounds having carboxyl groups bound to acyclic carbon atoms
30containing six-membered aromatic rings
CPC
A61K 31/192
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic, hydroximic acids
19Carboxylic acids, e.g. valproic acid
192having aromatic groups, e.g. sulindac, 2-arylpropionic acids, ethacrynic acid
A61K 9/20
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
20Pills, tablets, ; discs, rods
A61K 9/48
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
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
A61P 3/08
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
08for glucose homeostasis
C07C 57/30
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
CACYCLIC OR CARBOCYCLIC COMPOUNDS
57Unsaturated compounds having carboxyl groups bound to acyclic carbon atoms
30containing six-membered aromatic rings
Applicants
  • LIMINAL BIOSCIENCES LIMITED [GB]/[GB]
Inventors
  • GAGNON, Lyne
  • LAURIN, Pierre
  • CESARI, Frank
Agents
  • LAVERY, DE BILLY, L.L.P.
Priority Data
62/775,84905.12.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) USE OF SODIUM 2-(3-PENTYLPHENYL)ACETATE IN THE TREATMENT OF ALSTRÖM SYNDROME
(FR) UTILISATION DE 2-(3-PENTYLPHÉNYL)ACÉTATE DE SODIUM DANS LE TRAITEMENT DU SYNDROME D'ALSTRÖM
Abstract
(EN)
Methods, formulations/compositions and uses for improving at least one of liver histology, heart histology, kidney histology, adipose tissue histology and a metabolic parameter in a patient suffering from Alström syndrome are described, comprising administration of a pharmaceutical oral formulation comprising 2-(3-pentylphenyl)acetate or a pharmaceutically salt thereof and a pharmaceutically acceptable excipient at a daily dose of about 500 mg to about 1500 mg. The treatment is preferably for a period of more than 48 weeks and is not interrupted for 15 days or more.
(FR)
L'invention concerne des procédés, des formulations/compositions et des utilisations pour améliorer au moins l'une parmi l'histologie hépatique, l'histologie cardiaque, l'histologie rénale, l'histologie de tissu adipeux et un paramètre métabolique chez un patient souffrant d'un syndrome d'Alström, comprenant l'administration d'une formulation pharmaceutique orale comprenant du 2-(3-pentylphényle)acétate ou un sel pharmaceutiquement acceptable de celui-ci et un excipient pharmaceutiquement acceptable à une dose quotidienne d'environ 500 mg à environ 1500 mg. Le traitement est de préférence pendant une période de plus de 48 semaines et n'est pas interrompu pendant 15 jours ou plus.
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