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AR121415A1 - p38 KINASE INHIBITORS THAT REDUCE EXPRESSION OF DUX4 AND DOWNSTREAM GENES FOR USE IN THE TREATMENT OF FSHD - Google Patents

p38 KINASE INHIBITORS THAT REDUCE EXPRESSION OF DUX4 AND DOWNSTREAM GENES FOR USE IN THE TREATMENT OF FSHD

Info

Publication number
AR121415A1
AR121415A1 ARP180102885A ARP180102885A AR121415A1 AR 121415 A1 AR121415 A1 AR 121415A1 AR P180102885 A ARP180102885 A AR P180102885A AR P180102885 A ARP180102885 A AR P180102885A AR 121415 A1 AR121415 A1 AR 121415A1
Authority
AR
Argentina
Prior art keywords
dux4
fshd
treatment
kinase inhibitors
reduce expression
Prior art date
Application number
ARP180102885A
Other languages
Spanish (es)
Inventor
Angela Marie Cacace
Soto Luis Gustavo Alejandro Rojas
Iii Lorin A Thompson
Owen Brendan Wallace
Lucienne V Ronco
Ning Shen
Alan Scott Robertson
Aaron Nakwon Chang
Original Assignee
Fulcrum Therapeutics Inc
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.)
Filing date
Publication date
Application filed by Fulcrum Therapeutics Inc filed Critical Fulcrum Therapeutics Inc
Publication of AR121415A1 publication Critical patent/AR121415A1/en

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Reivindicación 1: Un inhibidor de la quinasa p38, en donde el inhibidor de la quinasa p38 posee la fórmula (1), o una sal farmacéuticamente aceptable del mismo; en el que la administración del inhibidor de la quinasa p38 reduce los niveles de expresión de un polipéptido DUX4 y/o un polipéptido codificado por un gen DUX4 corriente abajo en las células musculares del sujeto, caracterizado porque se usa para tratar la distrofia muscular facioescapulohumeral (FSHD), comprendiendo administrar a un sujeto que lo necesite, una cantidad eficaz de dicho compuesto.Claim 1: A p38 kinase inhibitor, wherein the p38 kinase inhibitor has the formula (1), or a pharmaceutically acceptable salt thereof; wherein the administration of the p38 kinase inhibitor reduces the expression levels of a DUX4 polypeptide and/or a polypeptide encoded by a downstream DUX4 gene in the muscle cells of the subject, characterized in that it is used to treat facioscapulohumeral muscular dystrophy ( FSHD), comprising administering to a subject in need thereof, an effective amount of said compound.

ARP180102885A 2017-10-05 2018-10-05 p38 KINASE INHIBITORS THAT REDUCE EXPRESSION OF DUX4 AND DOWNSTREAM GENES FOR USE IN THE TREATMENT OF FSHD AR121415A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762568673P 2017-10-05 2017-10-05

Publications (1)

Publication Number Publication Date
AR121415A1 true AR121415A1 (en) 2022-06-08

Family

ID=82401538

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP180102885A AR121415A1 (en) 2017-10-05 2018-10-05 p38 KINASE INHIBITORS THAT REDUCE EXPRESSION OF DUX4 AND DOWNSTREAM GENES FOR USE IN THE TREATMENT OF FSHD

Country Status (1)

Country Link
AR (1) AR121415A1 (en)

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AR121415A1 (en) p38 KINASE INHIBITORS THAT REDUCE EXPRESSION OF DUX4 AND DOWNSTREAM GENES FOR USE IN THE TREATMENT OF FSHD