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M

Marla Weetall

ptc therapeutics inc.
H指数:32
被引数:4,678

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MAPT splicing modulators reduce 4R tau and rescue tauopathy phenotypes in human neurons and in a mouse model

MAPT剪接调节剂可降低4Rtau并纠正tau病理表型，在人类神经元和在小鼠模型中

Science Translational Medicine
IF14.6
2026-08-190
PRE
M. Catarina Silva; Hannah Lindmeier; Paolo Pigini; Jennifer Laughlin; Yong Yu; Christie Morrill; Michael A. Arnold; Scott J. Barraza; Khalil Saadipour; Monal Dieterich; Angela Minnella; Lin-Ing Wang; Wencheng Li; Kausiki Datta; Nanjing Zhang; Jana Narasimhan; Christopher R. Trotta; Matthew G. Woll; Ellen M. Welch; Kellie Benzow; Kul Karanjeet; Steven Lotz; Taylor Bertucci; Sally Temple; Stephen J. Haggarty; Michael Koob; Jeffrey Trimmer; Marla Weetall; Elisabetta Morini
Exploratory Analysis of ELP1 Expression in Whole Blood From Patients With Familial Dysautonomia

家族性自主神经功能异常患者外周血中ELP1表达的可探索性分析

Annals of Clinical and Translational Neurology
IF3.9
2025-12-120
OAAI
Alejandra González-Duarte; Lucy Norcliffe-Kaufmann; Maria Luisa Cotrina; Zenith Khan; Kaia Dalamo; Patricio Millar Vernetti; Matthew Lawless; Elisabetta Morini; Monica Salani; Marla Weetall; Jana Narasimhan; Agostino G. Rocha; Susan A. Slaugenhaupt; Horacio Kaufmann
MOLECULAR AND BIOLOGY CORRELATIVE RESULTS FROM THE PHASE 1B TRIAL OF UNESBULIN (PTC596) WITH RADIOTHERAPY IN CHILDREN NEWLY-DIAGNOSED WITH DIFFUSE INTRINSIC PONTINE GLIOMA (DIPG) AND HIGH-GRADE GLIOMA (HGG): A REPORT FROM THE COLLABORATIVE NETWORK FOR NEURO-ONCOLOGY CLINICAL TRIALS (CONNECT)
NEURO-ONCOLOGY
IF13.4
2024-11-110
PRE
Lazow, Margot; Baxter, Patricia; Kumar, Shiva Senthil; Mohamad, Maya; Mackay, Alan; Stanek, Joseph; Rodriguez, Diana; Palmer, Joshua; Leach, James; Mikael, Leonie; Thomas, Diana; Fuller, Christine; Boue, Daniel; Pierson, Christopher; Breneman, John; Li, Xiao-Nan; Salloum, Ralph; Ashley, David; de Blank, Peter; Hwang, Eugene; Leary, Sarah; Plant, Ashley; Fisher, Michael; Chi, Susan; Murala, Mythili; Weetall, Marla; Rance, Mark; Baird, John; D'Silva, Dhiren; O'Keefe, Kylie; Leonard, Jeffrey; Stewart, Clinton; Jones, Chris; Mardis, Elaine; Fouladi, Maryam; Drissi, Rachid
Preclinical studies of gene replacement therapy for CDKL5 deficiency disorder
MOLECULAR THERAPY
IF12
2024-10-010
PRE
Voronin, Gregory; Narasimhan, Jana; Gittens, Jamila; Sheedy, Josephine; Lipari, Philip; Peters, Melinda; DeMarco, Steven; Cao, Liangxian; Varganov, Yakov; Kim, Min Jung; Pear, Lisset; Fotouh, Eman; Sinha, Supriya; Ray, Balmiki; Wu, Michael C.; Yalamanchili, Padmaja; Southgate, Christopher; Pick, Joseph; Saadipour, Khalil; Jung, Stephen; Lee, Jeanee; Mollin, Anna; Welch, Ellen M.; Wu, Zhijian; Weetall, Marla
Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models
HUMAN MOLECULAR GENETICS
IF3.2
2023-10-118
OAAI
Liu, Grace; Lipari, Philip; Mollin, Anna; Jung, Stephen; Teplova, Irina; Li, Wencheng; Ying, Lanqing; More, Vijay; Lennox, William; Yeh, Shirley; McGann, Eric; Moon, Young-Choon; Rice, Cari; Huarte, Eduardo; Gruszka, Barbara; Ray, Balmiki; Goodwin, Elizabeth; Buckendahl, Patricia; Yurkow, Edward; Braughton, Bruce; Narasimhan, Jana; Welch, Ellen; Voronin, Gregory; Weetall, Marla
UPDATED FINDINGS FROM THE PHASE IB STUDY OF UNESBULIN (PTC596) IN CHILDREN WITH NEWLY-DIAGNOSED DIFFUSE INTRINSIC PONTINE GLIOMA (DIPG) AND HIGH-GRADE GLIOMA (HGG): A REPORT FROM THE COLLABORATIVE NETWORK FOR NEURO-ONCOLOGY CLINICAL TRIALS (CONNECT)

新诊断的弥漫性内在性脑桥胶质瘤 (DIPG) 和高级别胶质瘤 (HGG) 儿童UNESBULIN (PTC596) IB期研究的最新发现: 来自神经肿瘤学临床试验合作网络 (CONNECT) 的报告

NEURO-ONCOLOGY
IF13.4
2023-06-120
OAAI
Lazow, Margot; Baxter, Patricia; Stanek, Joseph; Rodriguez, Diana; Kumar, Shiva Senthil; Palmer, Joshua; Leach, James; Mikael, Leonie; Thomas, Diana; Fuller, Christine; Boue, Daniel; Pierson, Christopher; Breneman, John; Li, Xiao-Nan; Salloum, Ralph; Ashley, David; De Blank, Peter; Hwang, Eugene; Leary, Sarah; Plant, Ashley; Fisher, Michael; Chi, Susan; Crabtree, Dorothy; Murali, Mythili; Weetall, Marla; Rance, Mark; Maliakal, Pius; Baird, John; D'Silva, Dhiren; O'Keefe, Kylie; Leonard, Jeffrey; Stewart, Clinton; Mardis, Elaine; Fouladi, Maryam; Drissi, Rachid
Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia
AMERICAN JOURNAL OF HUMAN GENETICS
IF8.1
2023-03-015
OAAI
Morini, Elisabetta; Chekuri, Anil; Logan, Emily M.; Bolduc, Jessica M.; Kirchner, Emily G.; Salani, Monica; Krauson, Aram J.; Narasimhan, Jana; Gabbeta, Vijayalakshmi; Grover, Shivani; Dakka, Amal; Mollin, Anna; Jung, Stephen P.; Zhao, Xin; Zhang, Nanjing; Zhang, Sophie; Arnold, Michael; Woll, Matthew G.; Naryshkin, Nikolai A.; Weetall, Marla; Slaugenhaupt, Susan A.
Emvododstat, a Potent Dihydroorotate Dehydrogenase Inhibitor, Is Effective in Preclinical Models of Acute Myeloid Leukemia
FRONTIERS IN ONCOLOGY
IF3.3
2022-02-0913
OAAI
Branstrom, Arthur; Cao, Liangxian; Furia, Bansri; Trotta, Christopher; Santaguida, Marianne; Graci, Jason D.; Colacino, Joseph M.; Ray, Balmiki; Li, Wencheng; Sheedy, Josephine; Mollin, Anna; Yeh, Shirley; Kong, Ronald; Sheridan, Richard; Baird, John D.; O'Keefe, Kylie; Spiegel, Robert; Goodwin, Elizabeth; Keating, Suzanne; Weetall, Marla
Small molecule splicing modifiers with systemic HTT-lowering activity

具有系统性HTT降低活性的小分子剪接修饰剂

NATURE COMMUNICATIONS
IF15.7
2021-12-1547
OAAI
Bhattacharyya, Anuradha; Trotta, Christopher R.; Narasimhan, Jana; Wiedinger, Kari J.; Li, Wencheng; Effenberger, Kerstin A.; Woll, Matthew G.; Jani, Minakshi B.; Risher, Nicole; Yeh, Shirley; Cheng, Yaofeng; Sydorenko, Nadiya; Moon, Young-Choon; Karp, Gary M.; Weetall, Marla; Dakka, Amal; Gabbeta, Vijayalakshmi; Naryshkin, Nikolai A.; Graci, Jason D.; Tripodi, Thomas, Jr.; Southwell, Amber; Hayden, Michael; Colacino, Joseph M.; Peltz, Stuart W.
Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent
MOLECULAR CANCER THERAPEUTICS
IF5.5
2021-07-2619
OAAI
Jernigan, Finith; Branstrom, Arthur; Baird, John D.; Cao, Liangxian; Dali, Mandar; Furia, Bansri; Kim, Min Jung; O'Keefe, Kylie; Kong, Ronald; Laskin, Oscar L.; Colacino, Joseph M.; Pykett, Mark; Mollin, Anna; Sheedy, Josephine; Dumble, Melissa; Moon, Young-Choon; Sheridan, Richard; Muehlethaler, Tobias; Spiegel, Robert J.; Prota, Andrea E.; Steinmetz, Michel O.; Weetall, Marla
Report of the National Institutes of Health SARS-CoV-2 Antiviral Therapeutics Summit

美国国立卫生研究院SARS-CoV-2抗病毒治疗峰会的报告

JOURNAL OF INFECTIOUS DISEASES
IF4.5
2021-06-1025
OAAI
Hall, Matthew D.; Anderson, James M.; Anderson, Annaliesa; Baker, David; Bradner, Jay; Brimacombe, Kyle R.; Campbell, Elizabeth A.; Corbett, Kizzmekia S.; Carter, Kara; Cherry, Sara; Chiang, Lillian; Cihlar, Tomas; de Wit, Emmie; Denison, Mark; Disney, Matthew; Fletcher, Courtney, V; Ford-Scheimer, Stephanie L.; Gotte, Matthias; Grossman, Abigail C.; Hayden, Frederick G.; Hazuda, Daria J.; Lanteri, Charlotte A.; Marston, Hilary; Mesecar, Andrew D.; Moore, Stephanie; Nwankwo, Jennifer O.; O'Rear, Jules; Painter, George; Saikatendu, Kumar Singh; Schiffer, Celia A.; Sheahan, Timothy P.; Shi, Pei-Yong; Smyth, Hugh D.; Sofia, Michael J.; Weetall, Marla; Weller, Sandra K.; Whitley, Richard; Fauci, Anthony S.; Austin, Christopher P.; Collins, Francis S.; Conley, Anthony J.; Davis, Mindy, I
Identification of a targetable KRAS-mutant epithelial population in non-small cell lung cancer
COMMUNICATIONS BIOLOGY
IF5.1
2021-04-1416
OAAI
Maroni, Giorgia; Bassal, Mahmoud A.; Krishnan, Indira; Fhu, Chee Wai; Savova, Virginia; Zilionis, Rapolas; Maymi, Valerie A.; Pandell, Nicole; Csizmadia, Eva; Zhang, Junyan; Storti, Barbara; Castano, Julio; Panella, Riccardo; Li, Jia; Gustafson, Corinne E.; Fox, Sam; Levy, Rachel D.; Meyerovitz, Claire, V; Tramontozzi, Peter J.; Vermilya, Kimberly; De Rienzo, Assunta; Crucitta, Stefania; Basseres, Daniela S.; Weetall, Marla; Branstrom, Art; Giorgetti, Alessandra; Ciampi, Raffaele; Del Re, Marzia; Danesi, Romano; Bizzarri, Ranieri; Yang, Henry; Kocher, Olivier; Klein, Allon M.; Welner, Robert S.; Bueno, Raphael; Magli, Maria Cristina; Clohessy, John G.; Ali, Azhar; Tenen, Daniel G.; Levantini, Elena
Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA
SCIENCE TRANSLATIONAL MEDICINE
IF14.6
2021-01-2765
OA
Kong, Lingling; Valdivia, David O.; Simon, Christian M.; Hassinan, Cera W.; Delestree, Nicolas; Ramos, Daniel M.; Park, Jae Hong; Pilato, Celeste M.; Xu, Xixi; Crowder, Melissa; Grzyb, Chloe C.; King, Zachary A.; Petrillo, Marco; Swoboda, Kathryn J.; Davis, Crystal; Lutz, Cathleen M.; Stephan, Alexander H.; Zhao, Xin; Weetall, Marla; Naryshkin, Nikolai A.; Crawford, Thomas O.; Mentis, George Z.; Sumner, Charlotte J.

研究方向

暂未获取到该数据
J
Josephine Sheedy
H 指数:14论文数:53被引数:1,240
J
Jana Narasimhan
H 指数:22论文数:53被引数:2,709
J
John D. Baird
H 指数:25论文数:145被引数:2,514
N
Nikolai A. Naryshkin
H 指数:21论文数:38被引数:2,525
E
Ellen Welch
H 指数:23论文数:106被引数:4,007
G
Gary M. Karp
H 指数:18论文数:66被引数:2,492
L
Liangxian Cao
H 指数:15论文数:57被引数:1,228
J
Joseph M. Colacino
H 指数:31论文数:100被引数:4,510
A
Anna Mollin
H 指数:10论文数:33被引数:1,374
Y
Young‐Choon Moon
H 指数:16论文数:64被引数:2,862