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

Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial

鞘内注射onasemnogene abeparvovec治疗初治脊髓性肌萎缩症患者:一项3期随机对照试验

Proud, Crystal M; Vũ, Dũng Chí; Wilmshurst, Jo M; Sanmaneechai, Oranee; Gulati, Sheffali; Xiong, Hui; Moreno, Hugo C; Tay, Stacey Kiat Hong; Thong, Meow-Keong; Born, Alfred Peter; Banzzatto Ortega, Adriana; Jong, Yuh-Jyh; Al-Muhaizea, Mohammad A; Lee, Anna W; Visootsak, Jeannie; Tauscher-Wisniewski, Sitra; Alecu, Iulian; Parlikar, Rutvick; Finkel, Richard S

Mpox Clade IIb Virus Introduction into Kinshasa, Democratic Republic of the Congo, July 2025

2025年7月,痘病毒IIb分支传入刚果民主共和国金沙萨。

Wawina-Bokalanga, Tony; Kinganda-Lusamaki, Eddy; Ngandu, Christian; Akil-Bandali, Prince; Kundey-Mafu, Jérémie; Ngombe, Nadege; Liesenborghs, Laurens; Paku-Tshambu, Princesse; Subissi, Lorenzo; Muswamba-Kayembe, Pauline-Chloé; Tessi-Mvutukulu, Samy; Santini-Mafuta, Jacques; Luakanda-Ndelemo, Gradi; Ntumba-Tshitenge, Olga; Keita, Mory; Cinkobu-Bualukengu, Israel; Lokilo-Lofiko, Emmanuel; Cikaya-Kankolongo, Fiston; Josaphat, Sikoti; Kacita, Cris; Lofungola, Adelar; Tete-Sitra, Judith; Lumembe-Numbi, Raphael; Mabika-Bope, Elzedek; Amuri-Aziza, Adrienne; Jansen, Daan; Makangara-Cigolo, Jean-Claude; Nkakulu, Jeanine; Boum, Yap; Ngongo, Ngashi; Tessema, Sofonias; Loman, Nick; O'Toole, Áine; Rimoin, Anne W; Akilimali, Pierre; Hoff, Nicole A; Kindrachuk, Jason; Ahuka-Mundeke, Steve; Peeters, Martine; Mwamba, Dieudonné; Vercauteren, Koen; Rambaut, Andrew; Muyembe-Tamfum, Jean-Jacques; Mbala-Kingebeni, Placide

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

鞘内注射 Onasemnogene Abeparvovec 用于坐姿、非行走型脊髓性肌萎缩症患者:I 期递增剂量研究 (STRONG)

Finkel, Richard S; Darras, Basil T; Mendell, Jerry R; Day, John W; Kuntz, Nancy L; Connolly, Anne M; Zaidman, Craig M; Crawford, Thomas O; Butterfield, Russell J; Shieh, Perry B; Tennekoon, Gihan; Brandsema, John F; Iannaccone, Susan T; Shoffner, John; Kavanagh, Sarah; Macek, Thomas A; Tauscher-Wisniewski, Sitra

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

Onasemnogene abeparvovec 用于携带两个 SMN2 基因拷贝且有患 1 型脊髓性肌萎缩症风险的无症状婴儿:III 期 SPR1NT 试验

Strauss, Kevin A; Farrar, Michelle A; Muntoni, Francesco; Saito, Kayoko; Mendell, Jerry R; Servais, Laurent; McMillan, Hugh J; Finkel, Richard S; Swoboda, Kathryn J; Kwon, Jennifer M; Zaidman, Craig M; Chiriboga, Claudia A; Iannaccone, Susan T; Krueger, Jena M; Parsons, Julie A; Shieh, Perry B; Kavanagh, Sarah; Tauscher-Wisniewski, Sitra; McGill, Bryan E; Macek, Thomas A

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

Onasemnogene abeparvovec 用于携带三个 SMN2 基因拷贝且有脊髓性肌萎缩症风险的无症状婴儿:III 期 SPR1NT 试验

Strauss, Kevin A; Farrar, Michelle A; Muntoni, Francesco; Saito, Kayoko; Mendell, Jerry R; Servais, Laurent; McMillan, Hugh J; Finkel, Richard S; Swoboda, Kathryn J; Kwon, Jennifer M; Zaidman, Craig M; Chiriboga, Claudia A; Iannaccone, Susan T; Krueger, Jena M; Parsons, Julie A; Shieh, Perry B; Kavanagh, Sarah; Wigderson, Melissa; Tauscher-Wisniewski, Sitra; McGill, Bryan E; Macek, Thomas A

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

针对罕见遗传性神经发育精神疾病的基因疗法

Davidson, Beverly L; Gao, Guangping; Berry-Kravis, Elizabeth; Bradbury, Allison M; Bönnemann, Carsten; Buxbaum, Joseph D; Corcoran, Gavin R; Gray, Steven J; Gray-Edwards, Heather; Kleiman, Robin J; Shaywitz, Adam J; Wang, Dan; Zoghbi, Huda Y; Flotte, Terence R; Tauscher-Wisniewski, Sitra; Tifft, Cynthia J; Sahin, Mustafa

The Current Landscape of Prevention Trials in Dementia

痴呆症预防试验的现状

Lee, Jonathan; Howard, Rebecca Sitra; Schneider, Lon S

Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

更正:Onasemnogene Abeparvovec疗法的临床试验和上市后安全性

Day, John W; Mendell, Jerry R; Mercuri, Eugenio; Finkel, Richard S; Strauss, Kevin A; Kleyn, Aaron; Tauscher-Wisniewski, Sitra; Tukov, Francis Fonyuy; Reyna, Sandra P; Chand, Deepa H

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

在接受onasemnogene abeparvovec治疗筛查的患者中检测腺相关病毒9型抗体

Day, John W; Finkel, Richard S; Mercuri, Eugenio; Swoboda, Kathryn J; Menier, Melissa; van Olden, Rudolf; Tauscher-Wisniewski, Sitra; Mendell, Jerry R

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

Onasemnogene Abeparvovec疗法的临床试验和上市后安全性

Day, John W; Mendell, Jerry R; Mercuri, Eugenio; Finkel, Richard S; Strauss, Kevin A; Kleyn, Aaron; Tauscher-Wisniewski, Sitra; Tukov, Francis Fonyuy; Reyna, Sandra P; Chand, Deepa H