A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and Summary of Product Characteristics (SMPCs); Communication from the ISTH SSC Working Group on Gene Therapy

UCL Discovery (University College London) 2026 Open access

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Authors: Mussert, Caroline MA, Miesbach, Wolfgang, Pratima Chowdary, Lillicrap, David, Mahlangu, Johnny, Flora Peyvandi, Steven W. Pipe, Srivastava, Alok, Jan Voorberg, Pierce, Glenn F, Radosław Kaczmarek, Paul A Batty
+3 more Cutica, Ilaria, Amit Chunilal Nathwani, Leebeek, Frank WG
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Fields of Study & Subjects

Hemophilia Treatment and Research Virus-based gene therapy research Platelet Disorders and Treatments

Publication details

Journal / source
UCL Discovery (University College London)
Publication date
1 February 2026
Language
EN