UY37918A - Inhibidores de la quinasa p38 que reducen la expresión de dux4 y de los genes corriente abajo para el tratamiento de fshd - Google Patents

Inhibidores de la quinasa p38 que reducen la expresión de dux4 y de los genes corriente abajo para el tratamiento de fshd

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Publication number
UY37918A
UY37918A UY0001037918A UY37918A UY37918A UY 37918 A UY37918 A UY 37918A UY 0001037918 A UY0001037918 A UY 0001037918A UY 37918 A UY37918 A UY 37918A UY 37918 A UY37918 A UY 37918A
Authority
UY
Uruguay
Prior art keywords
dux4
expression
fshd
treatment
reduce
Prior art date
Application number
UY0001037918A
Other languages
English (en)
Inventor
Marie Cacace Angela
Gustavo Alejandro Rojas Soto Luis
A Thompson Lorin Iii
Brendan Wallace Owen
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 UY37918A publication Critical patent/UY37918A/es

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
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    • A61K31/416—1,2-Diazoles condensed with carbocyclic ring systems, e.g. indazole
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    • A61K31/435—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
    • A61K31/4353—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
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    • A61K31/53—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
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Abstract

La invención se refiere a métodos y composiciones que incluyen agentes e inhibidores de la quinasa p38 que regulan la expresión de DUX4 y de los genes corriente abajo que incluyen, mas no se limitan a, ZSCAN4, LEUTX, PRAMEF2, TRIM43, MBD3L2, KHDC1L, RFPL2, CCNA1, SLC34A2, TPRX1, PRAMEF20, TRIM49, PRAMEF4, PRAME6, PRAMEF15, o ZNF280A. Se divulgan métodos útiles para tratar una enfermedad asociada con la expresión anormal de DUX4 y de los genes corriente abajo (por ejemplo, distrofia muscular facioescapulohumeral).
UY0001037918A 2017-10-05 2018-10-05 Inhibidores de la quinasa p38 que reducen la expresión de dux4 y de los genes corriente abajo para el tratamiento de fshd UY37918A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762568673P 2017-10-05 2017-10-05
US201762568754P 2017-10-05 2017-10-05
US201862682563P 2018-06-08 2018-06-08
US201862682565P 2018-06-08 2018-06-08

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UY37918A true UY37918A (es) 2019-04-30

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CA (4) CA3128468A1 (es)
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Families Citing this family (8)

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Publication number Priority date Publication date Assignee Title
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