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Inhibitor development in previously untreated patients with severe haemophilia: A comparison of included patients and outcomes between a clinical study and a registry‐based study
AIM: The aim of this study was to investigate whether a disease registry could serve as a suitable alternative to clinical studies to investigate safety of orphan drugs in children. METHODS: We used individual patient data from previously untreated patients (PUPs) with severe haemophilia A from the...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2020
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7586966/ https://www.ncbi.nlm.nih.gov/pubmed/32627880 http://dx.doi.org/10.1111/hae.14100 |