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C

Cristan Farmer

childrens hospital of philadelphia
H指数:45
被引数:5,966

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AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial

AAV9基因疗法在II型GM1神经节苷脂贮积症中的应用——一项I-II期临床试验

New England Journal of Medicine
IF78.5
2026-02-010
PRE
Lewis, Connor J.; D'souza, Precilla; Johnston, Jean M.; Acosta, Maria T.; Farmer, Cristan; Baker, Eva H.; Crowell, Anna; Mojica, Yoliann; Ashraf, Sumaiya; Joseph, Lisa; Vezina, Gilbert; Quezado, Zenaide; Yousef, Muhammad H.; Vardar, Zeynep; Shazeeb, Mohammed Salman; Corti, Manuela; Blackwood, Meghan; Coleman, Kirsten; Batista, Rita; Thurm, Audrey; De Boever, Erika; Gahl, William A.; Byrne, Barry J.; Flotte, Terence R.; Jiang, Xuntian; Gross, Amanda L.; Keeler, Allison M.; Gray-Edwards, Heather; Martin, Douglas R.; Sena-Esteves, Miguel; Tifft, Cynthia J.
Longitudinal Characterization of Males With X-Linked Creatine Transporter Deficiency: Final Results of a Multiyear Observational Study

X连锁肌酸转运蛋白缺乏症男性患者的纵向特征分析：多年观察研究的最终结果

Pediatric Neurology
IF2.1
2025-11-010
OAAI
Miller, Judith S.; Farmer, Cristan; Blair, Susan; Bianconi, Simona; Akshoomoff, Natacha; Anselm, Irina; Barshop, Bruce A.; Becker, Lindsey; Bennett, Amanda E.; Berry, Leandra N.; Berry-Kravis, Elizabeth M.; Bruchey, Aleksandra; Byars, Anna W.; Cimms, Tricia; Cecil, Kim M.; Covello, Maxine; Cubit, Laura S.; Das, Tanvi; Davis, Robert J.; Drye, Madison; Ficicioglu, Can; Fulton, John B.; Goin-Kochel, Robin P.; Guthrie, Whitney; Hallinan, Barbara E.; Hannah-Shmouni, Fady; Gustafson, Kathryn E.; Koeberl, Dwight D.; Longo, Nicola; Mamak, Eva; Mercimek-Andrews, Saadet; Michalak, Claire; Porter, Forbes D.; Rahhal, Samar; Rees, Linda; Spiridigliozzi, Gail A.; Stone, Caitlin; Sullivan, Nancy R.; Sutton, V. Reid; Thomas, Rebecca P.; Udhnani, Manisha; Waisbren, Susan; Xu, Michelle; Zhang, Lin; Brandabur, Melanie; Thurm, Audrey
GM1 gangliosidosis type II: Results of a 10-year prospective study
GENETICS IN MEDICINE
IF6.2
2024-07-012
OAAI
D'Souza, Precilla; Farmer, Cristan; Johnston, Jean M.; Han, Sangwoo T.; Adams, David; Hartman, Adam L.; Zein, Wadih; Huryn, Laryssa A.; Solomon, Beth; King, Kelly; Jordan, Christopher P.; Myles, Jennifer; Nicoli, Elena-Raluca; Rothermel, Caroline E.; Algarin, Yoliann Mojica; Huang, Reyna; Quimby, Rachel; Zainab, Mosufa; Bowden, Sarah; Crowell, Anna; Buckley, Ashura; Brewer, Carmen; Regier, Debra S.; Brooks, Brian P.; Acosta, Maria T.; Baker, Eva H.; Vezina, Gilbert; Thurm, Audrey; Tifft, Cynthia J.

研究方向

暂未获取到该数据
A
Audrey Thurm
H 指数:58论文数:301被引数:1.5万
C
Carlos A. Zarate
H 指数:121论文数:706被引数:5.2万
S
Susan E. Swedo
H 指数:79论文数:234被引数:2.1万
S
Somer Bishop
H 指数:49论文数:201被引数:1.3万
E
Elizabeth D. Ballard
H 指数:47论文数:210被引数:8,394
B
Bashkim Kadriu
H 指数:33论文数:78被引数:3,607
P
Peixiong Yuan
H 指数:41论文数:130被引数:7,442
A
Aaron J. Kaat
H 指数:26论文数:190被引数:2,957
L
Lisa Joseph
H 指数:15论文数:76被引数:1,268
L
Lawrence T. Park
H 指数:22论文数:47被引数:2,754