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Spark Therapeutics

Homepage URL: sparktx.com
Alternate name: Spark Therapeutics, Inc.
Description: company in Philadelphia, United States
Country: US
ROR: 00s93j442
Wikidata ID: entity/Q30269302
Crossref ID: 100017547
DOI: 10.13039/100017547
OpenAlex ID: F4320334149
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
2017 · Stephen Russell, Jean Bennett, et al. · The Lancet · 2,079
Analysis of the Prevalence of Microsatellite Instability in Prostate Cancer and Response to Immune Checkpoint Blockade
2018 · Wassim Abida, Michael L. Cheng, et al. · JAMA Oncology · 700
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Hepatitis C Guidance 2023 Update: American Association for the Study of Liver Diseases– Infectious Diseases Society of America Recommendations for Testing, Managing, and Treating Hepatitis C Virus Infection
2023 · Debika Bhattacharya, Andrew Aronsohn, et al. · Clinical Infectious Diseases · 398
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Laboratory Assessment of the Anticoagulant Activity of Direct Oral Anticoagulants
2016 · Bethany T. Samuelson, Adam Cuker, et al. · CHEST Journal · 354
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Atomic context-conditioned protein sequence design using LigandMPNN
2025 · Justas Dauparas, Gyu Rie Lee, et al. · Nature Methods · 350
Reversal of direct oral anticoagulants: Guidance from the Anticoagulation Forum
2019 · Adam Cuker, Allison Burnett, et al. · American Journal of Hematology · 320
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Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study
2023 · Volkan Granit, Michael Benatar, et al. · The Lancet Neurology · 313
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Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
2021 · Lindsey A. George, Paul E. Monahan, et al. · New England Journal of Medicine · 253
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Protein interaction networks revealed by proteome coevolution
2019 · Qian Cong, Ivan Anishchenko, et al. · Science · 240
International Guideline for Idiopathic Inflammatory Myopathy-Associated Cancer Screening: an International Myositis Assessment and Clinical Studies Group (IMACS) initiative
2023 · Alexander G. S. Oldroyd, Jeffrey P. Callen, et al. · Nature Reviews Rheumatology · 198
Update on clinical gene therapy for hemophilia
2018 · George Q. Perrin, Roland W. Herzog, et al. · Blood · 187
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Definition and Classification of Dystonia
2025 · Alberto Albanese, Kailash P. Bhatia, et al. · Movement Disorders · 165
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The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene
2018 · Daniel C. Chung, Mette Bertelsen, et al. · American Journal of Ophthalmology · 136
Cancer Susceptibility Mutations in Patients With Urothelial Malignancies
2019 · Maria I. Carlo, Vignesh Ravichandran, et al. · Journal of Clinical Oncology · 133
Silymarin in non-cirrhotics with non-alcoholic steatohepatitis: A randomized, double-blind, placebo controlled trial
2019 · Victor J. Navarro, Steven H. Belle, et al. · PLoS ONE · 133
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Management Strategies for CLN2 Disease
2017 · Ruth E. Williams, Heather R. Adams, et al. · Pediatric Neurology · 126
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In vitroandin vivorescue of aberrant splicing inCEP290-associated LCA by antisense oligonucleotide delivery
2016 · Alejandro Garanto, Daniel C. Chung, et al. · Human Molecular Genetics · 121
Binding and sensing diverse small molecules using shape-complementary pseudocycles
2024 · Linna An, Meerit Said, et al. · Science · 114
Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial
2023 · Steven W. Pipe, Peter Collins, et al. · Blood · 114
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Pathophysiology of dilated cardiomyopathy: from mechanisms to precision medicine
2024 · Marta Gigli, Davide Stolfo, et al. · Nature Reviews Cardiology · 106
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Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy
2022 · Nancy J Newman, Patrick Yu-Wai-Man, et al. · Brain · 101
Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium
2020 · Debra A. Thompson, Alessandro Iannaccone, et al. · Translational Vision Science & Technology · 98
Neoadjuvant triplet immune checkpoint blockade in newly diagnosed glioblastoma
2025 · Georgina V. Long, Elena Shklovskaya, et al. · Nature Medicine · 96
Degradation of CCNK/CDK12 is a druggable vulnerability of colorectal cancer
2021 · Sebastian M. Dieter, Christine Siegl, et al. · Cell Reports · 96
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Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer
2019 · Steven Pipe, Frank W.G. Leebeek, et al. · Molecular Therapy — Methods & Clinical Development · 95
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Works count: 299
Citation count: 12,110
H-index: 51
I10-index: 174
2-year mean citedness: 17.2
Awards count: 6
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