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European Journal of Pediatrics|June 2, 2026
Psychometric evaluation of Patient-Reported Outcomes Measurement Information System (PROMIS) in pediatric sickle cell disease in EuropeMaite E Houwing, Michiel A J Luijten, Madieke J Muntendam, et al.Frontiers in Genome Editing|November 24, 2025
Correction: Rewriting the script: gene therapy and genome editing for von Willebrand DiseaseAlastair Barraclough, Isabel Bär, Tirsa van Duijl, et al.Nederlands Tijdschrift Voor Geneeskunde|July 24, 2014
[Haemoglobinopathy in the 21st century: incidence, diagnosis and heel prick screening]Monique H Suijker, E A Lian Roovers, C J Karin Fijnvandraat, et al.Journal of Thrombosis and Haemostasis : JTH|November 7, 2024
The severe von Willebrand disease variant p.M771V leads to impaired anterograde trafficking of von Willebrand factor in patient-derived and base-edited endothelial colony-forming cellsIsabel Bär, Alastair Barraclough, Petra E Bürgisser, et al.Research and Practice in Thrombosis and Haemostasis|April 28, 2025
Plasma proteomics in patients with von Willebrand disease and hemophilia A highlights von Willebrand factor as main determinant of response to desmopressin treatmentJessica Del Castillo Alferez, Eva R Smit, Alexander B Meijer, et al.Thrombosis and Haemostasis|May 4, 2021
Emicizumab Dosing in Children and Adults with Hemophilia A: Simulating a User-Friendly and Cost-Efficient RegimenLaura H Bukkems, Kathelijn Fischer, Idske Kremer-Hovinga, et al.BMJ Open|June 27, 2023
DosEmi study protocol: a phase IV, multicentre, open-label, crossover study to evaluate non-inferiority of pharmacokinetic-guided reduced dosing compared with conventional dosing of emicizumab in people with haemophilia AAnouk Donners, Konrad van der Zwet, Antoine C G Egberts, et al.American Journal of Hematology|January 6, 2026
Effect of Red Blood Cell Transfusion on Inflammatory and Angiogenic Pathways in Patients With Sickle Cell DiseaseLydian A de Ligt, Sanjay R Thakoerdin, Maud Zwolsman, et al.Frontiers in Immunology|October 13, 2025
MHC class II presentation of FVIII-AnnexinA5 fusion proteins internalized by antigen presenting cellsMariarosaria Miranda, Michela Leoni, Carmen van der Zwaan, et al.Blood|August 19, 2021
Incidence and mortality rates of intracranial hemorrhage in hemophilia: a systematic review and meta-analysisAnne-Fleur Zwagemaker, Samantha C Gouw, Julie S Jansen, et al.Pageof 16