Jerry R. Mendell - Publications, H-index & Citations

Jerry R. Mendell

nationwide children's hospital

66H指数

290论文数

2.0W被引数

收录论文 49

发表时间

Five-Year Outcomes With Delandistrogene Moxeparvovec in Patients With Duchenne Muscular Dystrophy: A Phase 1/2a Study 达兰尼斯特罗根 Moxeparvovec 治疗杜氏肌营养不良症的五年结果：一项 1/2a 期研究

MUSCLE & NERVE

IF3.1

2026-06-03

OAAI

Jerry R. Mendell; Zarife Sahenk; Linda P. Lowes; Megan A. Iammarino; Lindsay N. Alfano; Craig M. McDonald; James E. Signorovitch; Jim Jin; Jing Li; Stefanie Mason; Louise R. Rodino-Klapac

A prospective observational study assessing the functional disease progression of LGMDR4, betasarcoglycan-related limb girdle muscular dystrophy 一项前瞻性观察研究，评估LGMDR4（β- sarcoglycan相关的 limb girdle muscular dystrophy）的功能性疾病进展。

JOURNAL OF NEUROMUSCULAR DISEASES

IF3.4

2025-05-13

PREAI

Megan A Iammarino; Natalie F Reash; Kiana Shannon; Maggie Dugan; Kelly Lehman; Alayne Meyers; Jerry R Mendell; Lindsay N Alfano; Linda P Lowes

Gross motor delays in infants and young boys with Duchenne muscular dystrophy 杜氏肌营养不良症婴儿和年幼男孩的大运动发育迟缓

JOURNAL OF NEUROMUSCULAR DISEASES

IF3.4

2024-12-08

OAAI

Lowes, Linda P.; Reash, Natalie F.; Iammarino, Megan A.; Connolly, Anne M.; Pietruszewski, Lindsay; Smith, Melissa A.; Peng, Jing; Steiner, Christopher L.; Tsao, Chang-Yong; Waldrop, Megan A.; Flanigan, Kevin M.; Chagat, Shannon; Meyer, Alayne P.; Mendell, Jerry R.; Alfano, Lindsay N.

ACMG STATEMENT Contributions from medical geneticists in clinical trials of genetic therapies: A points to consider statement of the American College of Medical Genetics and Genomics (ACMG)

GENETICS IN MEDICINE

IF6.2

2023-06-01

OAAI

Pena, Loren D. M.; Burrage, Lindsay C.; Enns, Gregory M.; Esplin, Edward D.; Harding, Cary; Mendell, Jerry R.; Niu, Zhiyv (Neal); Scharfe, Curt; Yu, Timothy; Koeberl, Dwight D.

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)鞘内注射Onasemnogene Abeparvovec用于坐着，非卧床的脊髓性肌萎缩症患者: I期递增剂量研究 (STRONG)

JOURNAL OF NEUROMUSCULAR DISEASES

IF3.4

2023-05-02

OAAI

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

Leveraging gene therapy to achieve long-term continuous or controllable expression of biotherapeutics

SCIENCE ADVANCES

IF12.5

2022-07-15

OAAI

Cripe, Timothy P.; Hutzen, Brian; Currier, Mark A.; Chen, Chun-Yu; Glaspell, Andrea M.; Sullivan, Grace C.; Hurley, Julia M.; Deighen, Mackenzie R.; Venkataramany, Akila S.; Mo, Xiaokui; Stanek, Joseph R.; Miller, Anthony R.; Wijeratne, Saranga; Magrini, Vincent; Mardis, Elaine R.; Mendell, Jerry R.; Chandler, Dawn S.; Wang, Pin-Yi

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

NATURE MEDICINE

IF50

2022-06-17

143

OAAI

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.

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

NATURE MEDICINE

IF50

2022-06-17

152

OAAI

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.

Safety, β-Sarcoglycan expression, and functional outcomes from systemic gene transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4

GENETICS IN MEDICINE

IF6.2

2022-03-01

OAAI

O'Rourke, Erin; Rodino-Klapac, Louise; Pozsgai, Eric; Lewis, Sarah; Griffin, Danielle; Meadows, Aaron; Lehman, Kelly; Church, Kathleen; Reash, Natalie; Iammarino, Megan; Powers, Brenna; Alfano, Lindsay; Lowes, Linda; Koenig, Erica; Neuhaus, Sarah; Li, Xiaoxi; Mendell, Jerry R.

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy (vol 44, pg 1109, 2021)

DRUG SAFETY

IF3.8

2021-12-23

OAAI

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.

Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue

NATURE MEDICINE

IF50

2021-10-04

PREAI

Thomsen, Gretchen; Burghes, Arthur H. M.; Hsieh, Caroline; Do, Janet; Chu, Binh T. T.; Perry, Stephanie; Barkho, Basam; Kaufmann, Petra; Sproule, Douglas M.; Feltner, Douglas E.; Chung, Wendy K.; McGovern, Vicki L.; Hevner, Robert F.; Conces, Miriam; Pierson, Christopher R.; Scoto, Mariacristina; Muntoni, Francesco; Mendell, Jerry R.; Foust, Kevin D.

Validity and Reliability of the Neuromuscular Gross Motor Outcome

PEDIATRIC NEUROLOGY

IF2.1

2021-09-01

PREAI

Alfano, Lindsay N.; Iammarino, Megan A.; Reash, Natalie F.; Powers, Brenna R.; Shannon, Kiana; Connolly, Anne M.; Waldrop, Megan A.; Noritz, Garey H.; Shell, Richard; Tsao, Chang-Yong; Flanigan, Kevin M.; Mendell, Jerry R.; Lowes, Linda P.

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

DRUG SAFETY

IF3.8

2021-08-12

OAAI

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.

Current Clinical Applications of In Vivo Gene Therapy with AAVs AAVs体内基因治疗的临床应用现状

MOLECULAR THERAPY

IF12

2021-02-01

452

OAAI

Mendell, Jerry R.; Al-Zaidy, Samiah A.; Rodino-Klapac, Louise R.; Goodspeed, Kimberly; Gray, Steven J.; Kay, Christine N.; Boye, Sanford L.; Boye, Shannon E.; George, Lindsey A.; Salabarria, Stephanie; Corti, Manuela; Byrne, Barry J.; Tremblay, Jacques P.

Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes

PEDIATRICS

IF6.4

2020-09-01

OAAI

Waldrop, Megan A.; Karingada, Cassandra; Storey, Mike A.; Powers, Brenna; Iammarino, Megan A.; Miller, Natalie F.; Alfano, Lindsay N.; Noritz, Garey; Rossman, Ian; Ginsberg, Matthew; Mosher, Kathryn A.; Broomall, Eileen; Goldstein, Jessica; Bass, Nancy; Lowes, Linda P.; Tsao, Chang-Yong; Mendell, Jerry R.; Connolly, Anne M.

Clinical development on the frontier: gene therapy for duchenne muscular dystrophy 前沿的临床发展: 杜氏肌营养不良症的基因治疗

EXPERT OPINION ON BIOLOGICAL THERAPY

IF4

2020-02-12

OAAI

Asher, Damon R.; Thapa, Khampaseuth; Dharia, Sachi D.; Khan, Navid; Potter, Rachael A.; Rodino-Klapac, Louise R.; Mendell, Jerry R.

AVXS-101 PHASE 3 STUDY IN SPINAL MUSCULAR ATROPHY TYPE 1

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

IF7.5

2019-11-14

PREAI

Day, John W.; Chiriboga, Claudia A.; Crawford, Thomas O.; Darras, Basil T.; Finkel, Richard S.; Connolly, Anne M.; Iannaccone, Susan T.; Kuntz, Nancy L.; Pena, Loren D. M.; Schultz, Meredith; Shieh, Perry B.; Smith, Edward C.; Kausar, Imran; Feltner, Douglas E.; Ogrinc, Francis G.; Ouyang, Haojun; Macek, Thomas A.; Kernbauer, Elaine; Muehring, Lynlee M.; L'Italien, James; Sproule, Douglas M.; Kaspar, Brian K.; Mendell, Jerry R.

From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1

PEDIATRIC NEUROLOGY

IF2.1

2019-11-01

125

OAAI

Al-Zaidy, Samiah A.; Mendell, Jerry R.

Intrathecal administration of AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): Phase 1/2A study (strong)

JOURNAL OF THE NEUROLOGICAL SCIENCES

IF3.2

2019-10-01

OAAI

Finkel, R. S.; Day, J. W.; Darras, B. T.; Kuntz, N. L.; Connolly, A. M.; Crawford, T. O.; Butterfield, R. J.; Shieh, P. B.; Tennekoon, G.; Iannaccone, S. T.; Meriggioli, M.; Ogrinc, F. G.; Kavanagh, S.; Kernbauer, E.; Whittle, J.; L'Italien, J.; Kaspar, B.; Sproule, D. M.; Spector, S. A.; Feltner, D. E.; Mendell, J. R.

Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): Global pivotal phase 3 study program (STR1VE-US, STR1VE-EU, STR1VE-AP)

JOURNAL OF THE NEUROLOGICAL SCIENCES

IF3.2

2019-10-01

PREAI

Mercuri, E.; Baranello, G.; Day, J. W.; Bruno, C.; Corti, S.; Chiriboga, C. A.; Crawford, T. O.; Darras, B. T.; Finkel, R. S.; Connolly, A. M.; Iannaccone, S. T.; Kuntz, N. L.; Masson, R.; Pena, L. D. M.; Baldinetti, F.; Schultz, M.; Shieh, P. B.; Smith, E. C.; Saito, K.; Scoto, M.; Spector, S. A.; Truncated, A. Authors; Sproule, D. M.; Mendell, J. R.; Muntoni, F.

研究方向