A brief overview of hemophilia A and B, including prevalence and epidemiology.
Videos: Rare Takes
Pediatric hematologist Shannon Walker, MD, answers key clinical questions surrounding treatment goals and selection, inhibitor screening, breakthrough bleeds, and more. These short, focused Q&As provide practical strategies for managing real-world patient scenarios.
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In this Guide
A brief overview of hemophilia A and B, including prevalence and epidemiology.
Inside hemophilia genetics, such as F8 and F9 mutations, inheritance patterns, and de novo variants. Plus, evolving approaches to females with hemophilia.
How to make an accurate diagnosis, including the role of coagulation screening, factor VIII and IX assays, inhibitor testing, and genetic evaluation.
A look at how FVIII/FIX deficiency contributes to variable disease severity and provides the biologic foundation for modern hemophilia therapies.
Factor deficiencies can disrupt clot formation, causing bleeding complications, arthropathy, the development of inhibitors, and long-term disease burden.
From acute bleeds and inhibitor considerations to long-term approaches.
Inside the evolving hemophilia treatment landscape, including AAV-based gene therapies, non-factor prophylaxis, and approaches that aim to reduce treatment burden.