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Benzylidenoguanidine derivatives and therapeutic use for the treatment of diseases due to poor protein folding
专利权人:
Medical Research Council;InFlectis BioScience
发明人:
GUEDAT, Philippe,BERTOLOTTI, Anne
申请号:
ES14700310
公开号:
ES2594378T3
申请日:
2014.01.10
申请国别(地区):
ES
年份:
2016
代理人:
摘要:
A compound of the formula (I), or a pharmaceutically acceptable salt thereof, ** Formula ** a tautomeric form thereof in which: R1 is alkyl, Cl, F or Br; R2 is H or F; R3 is selected from H and alkyl; R4 is selected from C (O) R6; R5 is H; or R4 and R5 are linked to form a heterocyclic group that is optionally substituted with one or more R10 groups; R6 is selected from R7, OR7 and NR8R9; R7, R8 and R9 are each independently selected from alkyl, cycloalkyl, aralkyl, cycloalkenyl, heterocyclyl and aryl, each of which is optionally substituted with one or more R10 groups; Each R10 is independently selected from halogen, OH, CN, COO-alkyl, aralkyl, SO2-alkyl, SO2-aryl COOH, CO alkyl, CO-aryl, NH2, NH-alkyl, N (alkyl) 2, CF3, alkyl and alkoxy; X and Z are each independently CR11, and Y is selected from CR11 and N; R11 is H or F; for use in the treatment of a disorder related to stress due to poor protein folding and, in particular, with the accumulation of misfolded proteins, chosen from Charcot Marie Tooth neuropathy; severe Dejerine-Sottas syndrome, retinal diseases, preferably retinitis pigmentosa, ciliopathies of the retina, macular degeneration or diabetic retinopathy and ALS.Un compuesto de la fórmula (I), o una sal farmacéuticamente aceptable del mismo,**Fórmula** una forma tautomérica del mismo en el que: R1 es alquilo, Cl, F o Br; R2 es H o F; R3 está seleccionado de entre H y alquilo; R4 está seleccionado de entre C(O)R6; R5 es H; o R4 y R5 están unidos para formar un grupo heterocíclico que opcionalmente se sustituye con uno o más grupos R10; R6 está seleccionado de entre R7, OR7 y NR8R9; R7, R8 y R9 están cada uno independientemente seleccionados de entre alquilo, cicloalquilo, aralquilo, cicloalquenilo, heterociclilo y arilo, cada uno de los cuales está sustituido opcionalmente con uno o más grupos R10; cada R10 está independientemente seleccionado de entre halógeno, OH, CN, COO-alquilo, aralquilo, SO2-alquilo, SO2-arilo COOH, CO alquilo, CO-arilo, NH2, NH-
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