Kathelijne Peerlinck

Coagulation factor activity and clinical bleeding severity in rare bleeding disorders: results from the European Network of Rare Bleeding Disorders

Flora Peyvandi, Roberta Palla, Marzia Menegatti et al. · Journal of Thrombosis and Haemostasis · 2012 · 488 citations · Full text

Thrombosis, Coagulation Factor Activity, Medicine +11

Comprehensive Rare Variant Analysis via Whole-Genome Sequencing to Determine the Molecular Pathology of Inherited Retinal Disease

Keren Carss, Gavin Arno, Marie Erwood et al. · The American Journal of Human Genetics · 2016 · 463 citations · Full text

Whole-genome Sequencing, Allelic Variant, Genetic Disorder +11

Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study

Steven W. Pipe, Midori Shima, Michaela Lehle et al. · The Lancet Haematology · 2019 · 373 citations · Full text

Haven 4, Hematology, Non-randomised Phase 3 +2

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

Steven W. Pipe, Frank W.G. Leebeek, Michael Recht et al. · New England Journal of Medicine · 2023 · 307 citations · Full text

Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia A

Corien L. Eckhardt, Alice S. van Velzen, Marjolein Peters et al. · Blood · 2013 · 214 citations · Full text