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1. (WO2018104172) COMPOUNDS FOR TREATING BARTH SYNDROME
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Pub. No.: WO/2018/104172 International Application No.: PCT/EP2017/081199
Publication Date: 14.06.2018 International Filing Date: 01.12.2017
IPC:
A61K 31/4015 (2006.01) ,A61K 31/45 (2006.01) ,A61K 31/4725 (2006.01) ,A61P 43/00 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
33
Heterocyclic compounds
395
having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
40
having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
4015
having oxo groups directly attached to the heterocyclic ring, e.g. piracetam, ethosuximide
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
33
Heterocyclic compounds
395
having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
435
having six-membered rings with one nitrogen as the only ring hetero atom
44
Non-condensed pyridines; Hydrogenated derivatives thereof
445
Non-condensed piperidines, e.g. piperocaine
45
having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
33
Heterocyclic compounds
395
having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
435
having six-membered rings with one nitrogen as the only ring hetero atom
47
Quinolines; Isoquinolines
472
Non-condensed isoquinolines, e.g. papaverine
4725
containing further heterocyclic rings
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43
Drugs for specific purposes, not provided for in groups A61P1/-A61P41/108
Applicants:
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) [FR/FR]; 3, rue Michel Ange 75016 PARIS, FR
UNIVERSITE DE BORDEAUX [FR/FR]; 35, place Pey Berland 33000 BORDEAUX, FR
ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES [FR/FR]; 47-83 Boulevard de l'Hôpital 75013 PARIS, FR
Inventors:
TRIBOUILLARD-TANVIER, Déborah; FR
LASSERRE, Jean-Paul; FR
DI RAGO, Jean-Paul; FR
Agent:
REGIMBEAU; 20, rue de Chazelles 75847 PARIS CEDEX 17, FR
Priority Data:
16306626.906.12.2016EP
Title (EN) COMPOUNDS FOR TREATING BARTH SYNDROME
(FR) COMPOSÉS POUR LE TRAITEMENT DU SYNDROME DE BARTH
Abstract:
(EN) The present invention relates to compounds and method for treating and/or preventing Barth syndrome as well as methods for identifying such compounds. In particular, the invention concerns compounds that inhibit the cytosolic protein synthesis for its use in the prevention and/or treatment of a disorder or disease associated to mitochondrial dysfunction such as Barth syndrome, wherein the mitochondrial dysfunction is associated with a deficiency in mature cardiolipin level in mitochondria membrane.
(FR) La présente invention concerne des composés et une méthode pour le traitement et/ou la prévention du syndrome de Barth ainsi que des procédés d'identification de tels composés. En particulier, l'invention concerne des composés qui inhibent la synthèse des protéines cytosoliques pour utilisation dans la prévention et/ou le traitement d'un trouble ou d'une maladie associé à un dysfonctionnement des mitochondries tel que le syndrome de Barth, le dysfonctionnement des mitochondries étant associé à une insuffisance de cardiolipine mature dans la membrane mitochondriale.
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)