CA3128043C - Traitement de ciliopathies a l'aide d'inhibiteurs de la glucosylceramide synthase (gcs) - Google Patents

Traitement de ciliopathies a l'aide d'inhibiteurs de la glucosylceramide synthase (gcs)

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Publication number
CA3128043C
CA3128043C CA3128043A CA3128043A CA3128043C CA 3128043 C CA3128043 C CA 3128043C CA 3128043 A CA3128043 A CA 3128043A CA 3128043 A CA3128043 A CA 3128043A CA 3128043 C CA3128043 C CA 3128043C
Authority
CA
Canada
Prior art keywords
compound
use according
syndrome
ciliopathy
halogen
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
CA3128043A
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English (en)
Other versions
CA3128043A1 (fr
Inventor
Oxana Ibraghimov-Beskrovnaya
Nikolay O. Bukanov
Herve Husson
Sarah E. MORENO
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Priority claimed from PCT/US2020/016588 external-priority patent/WO2020163337A1/fr
Publication of CA3128043A1 publication Critical patent/CA3128043A1/fr
Application granted granted Critical
Publication of CA3128043C publication Critical patent/CA3128043C/fr
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Abstract

La présente invention concerne un procédé de traitement d'une ciliopathie chez un sujet, le procédé comprenant l'administration au sujet d'une quantité efficace d'un composé quinuclidine. L'invention concerne également une composition pharmaceutique comprenant un composé quinuclidine destiné à être utilisé dans ledit procédé.
CA3128043A 2019-02-04 2020-02-04 Traitement de ciliopathies a l'aide d'inhibiteurs de la glucosylceramide synthase (gcs) Active CA3128043C (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962800993P 2019-02-04 2019-02-04
US62/800,993 2019-02-04
US201962851430P 2019-05-22 2019-05-22
US62/851,430 2019-05-22
PCT/US2020/016588 WO2020163337A1 (fr) 2019-02-04 2020-02-04 Traitement de ciliopathies à l'aide d'inhibiteurs de la glucosylcéramide synthase (gcs)

Publications (2)

Publication Number Publication Date
CA3128043A1 CA3128043A1 (fr) 2020-08-13
CA3128043C true CA3128043C (fr) 2026-03-24

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