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Projects
A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and summary of product characteristics: communication from the ISTH SSC working group on gene therapy.
Caroline M.A. Mussert 1 , Wolfgang Miesbach 2 , Pratima Chowdary 3 4 , David Lillicrap 5 , Johnny Mahlangu 6, Flora Peyvandi 7 8 , Steven W. Pipe 9, Alok Srivastava 10, Jan Voorberg 11, Glenn F. Pierce 12 , Radoslaw Kaczmarek 13 14 , Paul Batty 3 4 , Ilaria Cutica 15 , Amit Nathwani 3 4, Frank W.G. Leebeek 16 Gene therapy for hemophilia has recently been implemented as standard clinical care, requiring organizational and multistakeholder preparedness and clear guidelines. In addition to pharmaceutical summaries of product characteristics (SMPCs), various (inter)national guidance documents have been published. However, no guidance document or SMPC covers the entire gene therapy care pathway.