Current Outline of Exon Skipping Trials in Duchenne Muscular Dystrophy.
Current Outline of Exon Skipping Trials in Duchenne Muscular Dystrophy.
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DOI：
10.3390/genes13071241
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发表时间：
2022-07-14
期刊：
影响因子：
3.5
通讯作者：
Topaloglu, Haluk
中科院分区：
文献类型：
--
作者：
Eser, Gokce;Topaloglu, Haluk
杜氏肌营养不良症(DMD)的分子治疗已经进入临床实践。一种特殊的方法是外显子跳过，这是一种有着15年以上背景的方法。在早期工作的基础上，有几项前景看好的临床试验。其目的是能够启动足够的抗肌营养不良蛋白的产生，以改变进展速度，并创造临床向更好的转变。其中一些分子已经至少获得了卫生当局的有条件批准；然而，我们仍然需要新的积累数据。
Molecular treatments for Duchenne muscular dystrophy (DMD) are already in clinical practice. One particular means is exon skipping, an approach which has more than 15 years of background. There are several promising clinical trials based on earlier works. The aim is to be able to initiate the production of enough dystrophin to change the rate of progression and create a clinical shift towards the better. Some of these molecules already have received at least conditional approval by health authorities; however, we still need new accumulating data.
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影响因子：
168.9
作者：
Cirak, Sebahattin;Arechavala-Gomeza, Virginia;Guglieri, Michela;Feng, Lucy;Torelli, Silvia;Anthony, Karen;Abbs, Stephen;Garralda, Maria Elena;Bourke, John;Wells, Dominic J.;Dickson, George;Wood, Matthew J. A.;Wilton, Steve D.;Straub, Volker;Kole, Ryszard;Shrewsbury, Stephen B.;Sewry, Caroline;Morgan, Jennifer E.;Bushby, Kate;Muntoni, Francesco
通讯作者：
Muntoni, Francesco
DOI：
10.1038/s41431-020-0643-7
发表时间：
2020-09
期刊：
European journal of human genetics : EJHG
影响因子：
--
作者：
Fratter C;Dalgleish R;Allen SK;Santos R;Abbs S;Tuffery-Giraud S;Ferlini A
通讯作者：
Ferlini A
影响因子：
56.9
作者：
Goyenvalle, A;Vulin, A;Danos, O
通讯作者：
Danos, O
TREAT-NMD DMD 全球数据库：分析 7,000 多个杜氏肌营养不良症突变。
影响因子：
3.9
作者：
Bladen, Catherine L.;Salgado, David;Monges, Soledad;Foncuberta, Maria E.;Kekou, Kyriaki;Kosma, Konstantina;Dawkins, Hugh;Lamont, Leanne;Roy, Anna J.;Chamova, Teodora;Guergueltcheva, Velina;Chan, Sophelia;Korngut, Lawrence;Campbell, Craig;Dai, Yi;Wang, Jen;Barisic, Nina;Brabec, Petr;Lahdetie, Jaana;Walter, Maggie C.;Schreiber-Katz, Olivia;Karcagi, Veronika;Garami, Marta;Viswanathan, Venkatarman;Bayat, Farhad;Buccella, Filippo;Kimura, En;Koeks, Zaida;van den Bergen, Janneke C.;Rodrigues, Miriam;Roxburgh, Richard;Lusakowska, Anna;Kostera-Pruszczyk, Anna;Zimowski, Janusz;Santos, Rosario;Neagu, Elena;Artemieva, Svetlana;Rasic, Vedrana Milic;Vojinovic, Dina;Posada, Manuel;Bloetzer, Clemens;Jeannet, Pierre-Yves;Joncourt, Franziska;Diaz-Manera, Jordi;Gallardo, Eduard;Karaduman, A. Ayse;Topaloglu, Haluk;El Sherif, Rasha;Stringer, Angela;Shatillo, Andriy V.;Martin, Ann S.;Peay, Holly L.;Bellgard, Matthew I.;Kirschner, Jan;Flanigan, Kevin M.;Straub, Volker;Bushby, Kate;Verschuuren, Jan;Aartsma-Rus, Annemieke;Beroud, Christophe;Lochmueller, Hanns
通讯作者：
Lochmueller, Hanns
影响因子：
3.7
作者：
Beekman C;Janson AA;Baghat A;van Deutekom JC;Datson NA
通讯作者：
Datson NA