日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial

鞘内注射onasemnogene abeparvovec治疗既往接受过治疗的脊髓性肌萎缩症患者:一项3b期开放标签试验

Kwon, Jennifer M; Munell, Francina; Le Goff, Laure; Yuge, Kotaro; Kato, Tamaki; Cances, Claude; De Waele, Liesbeth; Woodcock, Ian R; Mercuri, Eugenio M; Proud, Crystal M; Darras, Basil T; Hayes, Leslie H; Oskoui, Maryam; Visootsak, Jeannie; Williams, Gemma; Ilić, Andreja; Yang, Lina; van der Pol, W Ludo

Author Correction: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial

作者更正:鞘内注射onasemnogene abeparvovec治疗既往接受过治疗的脊髓性肌萎缩症患者:一项3b期开放标签试验

Kwon, Jennifer M; Munell, Francina; Le Goff, Laure; Yuge, Kotaro; Kato, Tamaki; Cances, Claude; De Waele, Liesbeth; Woodcock, Ian R; Mercuri, Eugenio M; Proud, Crystal M; Darras, Basil T; Hayes, Leslie H; Oskoui, Maryam; Visootsak, Jeannie; Williams, Gemma; Ilić, Andreja; Yang, Lina; van der Pol, W Ludo

Liver Steatosis in Induced Hepatocytes From Carriers of Spinal Muscular Atrophy

脊髓性肌萎缩症携带者诱导肝细胞的肝脂肪变性

Sun, Lingyu; Leow, Damien Meng Kiat; Wang, Loo Chien; Eio, Michelle Yating; Koh, Hiromi W L; Khong, Zi Jian; Narayanan, Gunaseelan; Teo, Aloysius Kai Soon; Giadone, Richard; Sobota, Radoslaw M; Ng, Shi Yan; Teo, Adrian Kee Keong; Ong, Wei Yi; Rubin, Lee L; Darras, Basil T; Yeo, Crystal J J

The essential genome of Plasmodium knowlesi reveals determinants of antimalarial susceptibility.

诺氏疟原虫的基本基因组揭示了抗疟药物敏感性的决定因素

Elsworth Brendan, Ye Sida, Dass Sheena, Tennessen Jacob A, Sultana Qudseen, Thommen Basil T, Paul Aditya S, Kanjee Usheer, Grüring Christof, Ferreira Marcelo U, Gubbels Marc-Jan, Zarringhalam Kourosh, Duraisingh Manoj T

An all-in-one pipeline for the in vitro discovery and in vivo testing of Plasmodium falciparum malaria transmission blocking drugs.

用于体外发现和体内测试恶性疟原虫疟疾传播阻断药物的一体化流程

Brancucci Nicolas M B, Gumpp Christin, van Gemert Geert-Jan, Yu Xiao, Passecker Armin, Nardella Flore, Thommen Basil T, Chambon Marc, Turcatti Gerardo, Halby Ludovic, Blasco Benjamin, Duffey Maëlle, Arimondo Paola B, Bousema Teun, Scherf Artur, Leroy Didier, Kooij Taco W A, Rottmann Matthias, Voss Till S

Analysis of human urinary extracellular vesicles reveals disordered renal metabolism in myotonic dystrophy type 1

对人类尿液细胞外囊泡的分析揭示了1型强直性肌营养不良症患者的肾脏代谢紊乱

Preeti Kumari,Lauren M Sullivan,Zhaozhi Li,E Parker Conquest,Elizabeth Cornforth,Rojashree Jayakumar,Ningyan Hu,J Alexander Sizemore,Brigham B McKee,Robert R Kitchen,Paloma González-Pérez,Constance Linville,Karla Castro,Hilda Gutierrez,Soleil Samaan,Elise L Townsend,Basil T Darras,Seward B Rutkove,Susan T Iannaccone,Paula R Clemens,Araya Puwanant,Sudeshna Das,Thurman M Wheeler

Spinal Muscular Atrophy Functional Composite Score Revised (SMA-FCR) in Untreated and Nusinersen-Treated Patient Cohorts

脊髓性肌萎缩症功能综合评分修订版(SMA-FCR)在未治疗和接受Nusinersen治疗的患者队列中的比较

Pasternak, Amy; McDermott, Michael P; Montes, Jacqueline; Glanzman, Allan M; Coratti, Giorgia; Dunaway Young, Sally; Duong, Trinh Tina; Martens, William B; Day, John W; Zolkipli-Cunningham, Zarazuela; Sansone, Valeria Ada; D'Amico, Adele; Messina, Sonia; Bruno, Claudio; Mercuri, Eugenio; De Vivo, Darryl C; Darras, Basil T

Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study

静脉注射奥那西姆诺基因阿贝帕沃韦治疗脊髓性肌萎缩症患儿的安全性和有效性:3b期SMART研究

McMillan, Hugh J; Baranello, Giovanni; Farrar, Michelle A; Zaidman, Craig M; Moreno, Teresa; De Waele, Liesbeth; Jong, Yuh-Jyh; Laugel, Vincent; Quijano-Roy, Susana; Mercuri, Eugenio; Chien, Yin-Hsiu; Straub, Volker; Darras, Basil T; Seibert, Julia; Bernardo Escudero, Roberto; Alecu, Iulian; Freischläger, Frank; Muntoni, Francesco

Exome and Genome Sequencing to Diagnose the Genetic Basis of Neonatal Hypotonia: An International Consortium Study

利用外显子组和基因组测序诊断新生儿肌张力低下的遗传基础:一项国际联盟研究

Morton, Sarah U; Costain, Gregory; French, Courtney E; Wakeling, Emma; Szuto, Anna; Christodoulou, John; Cohn, Ronald; Darras, Basil T; Wojcik, Monica H; D'Gama, Alissa M; Dowling, James J; Lunke, Sebastian; Muntoni, Francesco; Raymond, Lucy; Rowitch, David; Beggs, Alan H; Stark, Zornitza; Agrawal, Pankaj B

Longitudinal Assessment of 4-Year HFMSE Changes in SMA II and III Patients Treated With Nusinersen

对接受诺西那生治疗的SMA II型和III型患者进行4年HFMSE评分变化的纵向评估

Coratti, Giorgia; Bovis, Francesca; Pane, Marika; Pasternak, Amy; Albamonte, Emilio; Mizzoni, Irene; Glanzman, Allan M; Morando, Simone; Montes, Jacqueline; Cavallina, Ilaria; Dunaway Young, Sally; Duong, Tina; Rolle, Enrica; Civitello, Matthew; De Sanctis, Roberto; Bravetti, Chiara; Ricci, Federica; Gadaleta, Giulio; Mongini, Tiziana; Sframeli, Maria; Pera, Maria Carmela; Messina, Sonia; D'Amico, Adele; Catteruccia, Michela; Brolatti, Noemi; Hirano, Michio; Zolkipli-Cunningham, Zarazuela; Darras, Basil T; Bertini, Enrico; Bruno, Claudio; Day, John; Sansone, Valeria A; Finkel, Richard S; Mercuri, Eugenio