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Recommendations for a Minimum Data Set for Monitoring Gene Therapy in Hemophilia: Communication from the ISTH SSC Working Group on Gene Therapy.

Wolfgang Miesbach,Barbara Konkle,Pratima Chowdary,Radoslaw Kaczmarek,Frank Leebeek,Johnny Mahlangu,Michael Makris,Steven W. Pipe,Alok Srivastava,Jan Voorberg,Glenn F. Pierce,Flora Peyvandi

JOURNAL OF THROMBOSIS AND HAEMOSTASIS（2024）

Cited 1|Views33
Key words
gene therapy,hemophilia,minimal data,monitoring,registry
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