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1. (WO2019046755) METHODS FOR TREATING MUSCULAR DYSTROPHY
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公布号: WO/2019/046755 国际申请号: PCT/US2018/049151
公布日: 07.03.2019 国际申请日: 31.08.2018
国际专利分类:
A61K 31/00 (2006.01) ,C12N 15/113 (2010.01)
A 人类生活必需
61
医学或兽医学;卫生学
K
医用、牙科用或梳妆用的制剂
31
含有机有效成分的医药配制品
C 化学;冶金
12
包括微生物或酶的生物化学、微生物学、酶学,它们的制备;用其来合成化合物或组合物;涉及微生物或酶的测定或检验方法;变异或遗传工程
N
微生物或酶;其组合物;繁殖、保藏或维持微生物;变异或遗传工程;培养基
15
突变或遗传工程;遗传工程涉及的DNA或RNA,载体(如质粒)或其分离、制备或纯化;所使用的宿主
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
调节基因表达的非编码核酸,如反义寡核苷酸
申请人:
SAREPTA THERAPEUTICS, INC. [US/US]; 215 First Street Cambridge, Massachusetts 02142, US
发明人:
KAYE, Edward M.; US
代理人:
STEFFE, Eric K.; US
STERNE, Robert Greene; US
GOLDSTEIN, Jorge A.; US
CORNWELL, David K.S.; US
DURKIN, Tracy-Gene G.; US
ESMOND, Robert W.; US
CIMBALA, Michele A.; US
FEATHERSTONE, Donald J.; US
RAY, Michael B.; US
MILLONIG, Robert C.; US
LEE, Michael Q.; US
SOKOHL, Robert E.; US
LONGSWORTH, Gaby L.; US
COVERT, John M.; US
ELLISON, Eldora L.; US
SHEA JR., Timothy J.; US
BANOWIT, Donald R.; US
EISENBERG, Jason D.; US
HELVEY, Jeffrey T.; US
RYGIEL, Mark W.; US
JACKMAN, Peter A.; US
BODENSTEIN, Matthew; US
WRIGHT, Jon E.; US
YONAN, Daniel E.; US
HOLOUBEK, Michelle K.; US
SPECHT, Michael D.; US
HICKS, Ross; US
CALVO, Paul A.; US
ROSE-GILLENTINE, Marsha; US
COLLER III, Richard D.; US
BEZOS, Salvador M.; US
TUMINARO, Jonathan; US
VARUGHESE, Dennies; US
STERLING, Deborah A.; US
MUTSCHELKNAUS, Joseph; US
PICKARD, Byron L.; US
FRUEAUF, Jeremiah B.; US
NANNENGA-COMBS, Bonnie; US
KIM, Ji-Eun; US
DESAI, Nirav N.; US
VIRA, Chandrika; US
CAMARCE, Christian; US
CONKLIN, Kyle; US
POWERS III, R. Wilson; US
GAJEWSKI, Daniel; US
LAROCK, Adam; US
优先权数据:
62/553,09431.08.2017US
62/565,82429.09.2017US
62/725,12930.08.2018US
标题 (EN) METHODS FOR TREATING MUSCULAR DYSTROPHY
(FR) MÉTHODES PERMETTANT DE TRAITER LA DYSTROPHIE MUSCULAIRE
摘要:
(EN) The present disclosure provides, among other things, improved compositions and methods for treating muscular dystrophy. For example, the disclosure provides methods for treating Duchenne muscular dystrophy patients having a mutation in the DMD gene that is amenable to exon 53 skipping by administering an effective amount of golodirsen.
(FR) La présente invention concerne, entre autres, des compositions et méthodes améliorées permettant de traiter la dystrophie musculaire. À titre d'exemple, l'invention concerne des méthodes permettant de traiter des patients atteints de dystrophie musculaire de Duchenne ayant une mutation du gène DMD qui est susceptible de sauter l'exon 53 par administration d'une quantité efficace de golodirsen.
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指定国: 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
非洲地区知识产权组织 (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
欧亚专利局 (AM, AZ, BY, KG, KZ, RU, TJ, TM)
欧洲专利局 (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)
非洲知识产权组织 (OAPI) (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
公布语言: 英语 (EN)
申请语言: 英语 (EN)