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Published byIvan Rachman Modified over 6 years ago
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Hemophilia Updates: Incorporating New Concepts Into Practice
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Introduction
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SIPPET Study
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SIPPET Study Results
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Interpreting the Results
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MASAC Guidance
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WFH Guidance
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Emphasis From the WFH
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Further Notes on the SIPPET Study
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Further Notes on the SIPPET Study (cont)
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Nonreplacement Treatment Approaches
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Emicizumab
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Results of the Extended Emicizumab Trial
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Fitusiran
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Results of the Phase 1 Fitusiran Trial
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Further Thoughts on Novel Approaches
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Overview of EHL Factor Concentrates Available in the United States
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Clinical Experience With the EHL Factor Products
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Clinical Experience With the EHL Factor Products (cont)
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EHL Factor Products: Real-World Data
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EHL Factor Products: Real-World Data (cont)
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Gene Therapy
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Gene Therapy: Preliminary Results
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Concluding Remarks
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Abbreviations
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Abbreviations (cont)
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