ME01969B - Sredstva za lijecenje poremecaja koji ukljucuju modulaciju receptora rijanodina - Google Patents

Sredstva za lijecenje poremecaja koji ukljucuju modulaciju receptora rijanodina

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Publication number
ME01969B
ME01969B MEP-2014-147A MEP2014147A ME01969B ME 01969 B ME01969 B ME 01969B ME P2014147 A MEP2014147 A ME P2014147A ME 01969 B ME01969 B ME 01969B
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diseases
disorders
accordance
group
pharmaceutical preparation
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Jean-Louis Peglion
Jiaming Yan
Sandro Belvedere
Yael Webb
Marc Bertrand
Nicole Villeneuve
Andrew R Marks
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Servier Lab
Armgo Pharma Inc
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Publication of ME01969B publication Critical patent/ME01969B/me

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Claims (23)

1. Spoj, naznačen time, da je predstavljen strukturom Formule (I):pri čemuR je COOH;i njihove farmaceutski prihvatljive soli.
2. Spoj u skladu s patentnim zahtjevom 1, naznačen time, da je u obliku soli s farmaceutski prihvatljivom kiselinom ili lužinom.
3. Spoj u skladu s patentnim zahtjevom 2, naznačen time, da je sol odabrana iz skupine koja se sastoji od natrija, kalija, magnezija, hemifumarata, hidroklorida i hidrobromida.
4. Spoj u skladu s patentnim zahtjevom 1, naznačen time, da je odabran izskupine koja se sastoji od:
5. Spoj u skladu s patentnim zahtjevom 1, naznačen time, da je predstavljenstrukturnom Formulom (1):ili njihovom farmaceutski prihvatljivom soli.
6. Spoj u skladu s patentnim zahtjevom 5, naznačen time, da je u obliku solis farmaceutski prihvatljivom kiselinom ili lužinom.
7. Spoj u skladu s patentnim zahtjevom 6, naznačen time, da je sol odabranaiz skupine koja se sastoji od natrija, kalija, magnezija, hemifumarata, hidroklorida i hidrobromida.
8. Spoj u skladu s patentnim zahtjevom 7, naznačen time, da je sol natrijeva sol.
9. Spoj u skladu s patentnim zahtjevom 7, naznačen time, da je sol hemifumaratna sol.
10. Farmaceutski pripravak koji sadrži spoj u skladu s patentnim zahtjevima 1-9, naznačena time, da je u kombinaciji s jednim ili više farmaceutski prihvatljivih ekscipijensa ili nosača.
11. Farmaceutski pripravak u skladu s patentnim zahtjevom 10, naznačen time, da se koristi u liječenju ili prevenciji stanja odabranih is skupine koja se sastoji od srčanih poremećaja i oboljenja, mišićnog umora, poremećaja i bolesti muskuloskeletalnog sustava, poremećaja i bolesti središnjeg živčanog sustava (CNS), kognitivne disfunkcije, nervno-mišićnih poremećaja i oboljenja, poremećaja i bolesti kostiju, tumorske kaheksije, maligne hipertermije, dijabetesa, iznenadne srčane smrti i sindroma iznenadne dječje smrti ili za poboljšanje kognitivne funkcije.
12. Farmaceutski pripravak u skladu s patentnim zahtjevom 10, za uporabu u liječenju ili prevenciji stanja u skladu s patentnim zahtjevom 11, naznačen time, da su srčani poremećaji ili oboljenja odabrani iz skupine koja se sastoji od poremećaja ili bolesti nepravilnog rada srca poput primjerice atrijske i ventrikularne aritmije, atrijske i ventrikularne fibrilacije, atrijske i ventrikularne tahiaritmije, atrijske i ventrikularne tahikardije, kateholaminergičke polimorfne ventrikularne tahikardije (CPVT) te njihovih varijanti izazvanih usljed tjelovježbe; poremećaja i bolesti nepravilnog rada srca izazvanog usljed tjelovježbe; srčanog zatajenja; kongestivnog zatajenja srca; kroničnog zatajenja srca; akutnog zatajenja srca; sistoličkog zatajenja srca; dijastoličkog zatajenja srca; akutne dekompenzacije srca; srčane ishemije/reperfuzijska ozljeda (I/R); kronične opstruktivne plućne bolesti; I/R ozljede usljed srčane angioplastije ili usljed trombolize za liječenje infarkta miokarda (MI); te visokog krvanog tlaka.
13. Farmaceutski pripravak u skladu s patentnim zahtjevom 10 za uporabu u liječenju ili prevenciji u skladu s patentnim zahtjevom 11, naznačen time, da je mišićni zamor uzrokovan bolešću, poremećajem ili stanjem skeletnih mišića.
14. Farmaceutski pripravak u skladu s patentnim zahtjevom 10 za uporabu u liječenju ili prevenciji u skladu s patentnim zahtjevom 11, naznačen time, da je muskuloskeletalni poremećaj, bolest ili stanje odabrano iz skupine koja se sastoji zamora skeletnih mišića izazvanog usljed tjelovježbe; zamora mišića izazvanog usljed produljene ili intenzivne tjelovježbe; kongenitalne miopatije; mišične distrofije poput primjerice Duchenne-ove distrofije mišića (DMD), Becker-ove distrofije mišića (BMD), Limb-Girdle distrofije mišića (LGMD), facioskapulohumeralne mišićne distrofije, miotonične mišične distrofije, kongenitalne mišične distrofije (CMD), distalne mišične distrofije, Emery-Dreifuss-ove mišične distrofije i okulofaringealne mišićne distrofije; spinalne mišićne atrofije (SMA), spinalne i bulbarne mišićne atrofije (SBMA), Zamora mišića povezanog sa starosnom dobi; sarkopenije, bolesti središnje jezgre; tumorske kaheksije; poremećaje mjehura i inkontinencije.
15. Farmaceutski pripravak u skladu s patentnim zahtjevom 10 za uporabu u liječenju ili prevenciji u skladu s patentnim zahtjevom 11, naznačen time, da su poremećaj ili bolesti središnjeg živčanog sustava (CNS) odabrani iz skupine koja se sastoji od Alzheimer-ove bolesti (AD), neuropatije, napadaja, Parkinson-ove bolesti (PD) i Huntington-ove bolesti (HD); i nervno-mišićnih poremećaja i bolesti koje su odabrane iz skupine koja se sastoji od spinocerebelarne ataksije (SCA) i amiotrofna lateralna skleroza (ALS, Lou Gehrig-ova bolest).
16. Farmaceutski pripravak u skladu s patentnim zahtjevom za uporabu u liječenju ili prevenciji u skladu s patentnim zahtjevom 11, naznačen time, da je kognitivna disfunkcija povezana sa stresom ili sa starosnom dobi, ili gdje kognitivna funkcija treba biti poboljšana zbog kratkog pamćenja, dugotrajnog pamćenja, pažnje ili učenja, ili gdje je kognitivna disfunkcija povezana s bolesti ili poremećajem koji je odabran iz skupine koja se sastoji od Alzheimer-ove bolesti (AD), poremećaja pažnje uzrokovanog hiperaktivnošću (ADHD), poremećaja autističnog spektra (ASD), generaliziranog anksioznog poremećaja (GAD), opsesivno-kompulzivnog poremećaja (OCD), Parkinsonove bolesti (PD), post-traumatskog stresnog poremećaja (PTSD), Schizophrenije, bipolarnog poremećaja i unipolarne velike depresije.
17. Farmaceutski pripravak u skladu s patentnim zahtjevom 10 za uporabu u liječenju ili prevenciji u skladu s patentnim zahtjevom 11, naznačen time, da je stanje tumorska kaheksija.
18. Farmaceutski pripravak u skladu s patentnim zahtjevom 17, naznačen time, da je tumorska kaheksija uzrokovana usljed metastaziranja tumora kosti.
19. Spoj u skladu s bilo kojim od patentnih zahtjeva 1-9, ili njihove soli, naznačen time, da se koristi za liječenje ili prevenciju stanja odabranih iz skupine koja se sastoji od srčanih poremećaja ili oboljenja, zamora mišića, poremećaja muskuloskeletalnog sustava, poremećaja i bolesti središnjeg živčanog sustava (CNS), kognitivne disfunkcije, nervno-mišićnih poremećaja i bolesti, poremećaja i bolesti kosti, tumorske kaheksije, maligne hipertermije, dijabetesa, iznenadne srčane smrti i sindroma iznenadne dječje smrti ili za poboljšanje kognitivne funkcije.
20. Farmaceutski pripravak u skladu s patentnim zahtjevom 10 u kombinaciji s antisens-oligonukleotidom (AO) koji je specifičan za upletanje odsječka u ciljanoj mRNK za bolje preskakanje egzona u rečenoj ciljanoj mRNK, naznačen time, da se koristi za uporabu u liječenju ili prevenciji u skladu s patentnim zahtjevom 11.
21. Farmaceutski pripravak u skladu s patentnim zahtjevom 20, naznačen time, da se koristi u liječenju Duchenne-ove mišićne distrofije (DMD), pri čemu je antisens-oligonukleotid (AO) specifičan za upletanje odsječka od najmanje jednog od egzona DMD gena.
22. Farmaceutski pripravak u skladu s patentnim zahtjevom 21, naznačen time, da je antisens-oligonukleotid specifičan za upletanje odsječka egzona 23, 45, 44, 50, 51, 52 i /ili 53 DMD gena.
23. Postupak priprave u skladu s bilo kojim od patentnih zahtjeva 1-9, naznačen time, da se sastoji od koraka reakcije spojeva formule sa spojem formulesa spojem formulepri čemu Ra je COOR1 ili CN; R1 je C1-C4 alkil, a L je odlazeća skupina kako bi se dobio spoj formule:ipretvaranjem skupine Ra u skupinu R kako bi se dobio spoj formule (I).
MEP-2014-147A 2012-04-18 2013-04-17 Sredstva za lijecenje poremecaja koji ukljucuju modulaciju receptora rijanodina ME01969B (me)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261625890P 2012-04-18 2012-04-18
EP12167732.2A EP2708535A1 (en) 2012-05-11 2012-05-11 Agents for treating disorders involving modulation of ryanodine receptors
EP20130164051 EP2653466B1 (en) 2012-04-18 2013-04-17 Agents for treating disorders involving modulation of ryanodine receptors

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ME01969B true ME01969B (me) 2015-05-20

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