Stay informed with the latest updates for the inheritable blood disorders community.

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Investigational Sub-Q Von Willebrand Disease Therapy Put on Fast Track
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Trial Updates for Investigational Hemophilia A Gene Therapy Shared at ASH…
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A person pointing at their arm with purple spots
Sanofi Announces Positive Data for Its Investigational ITP Therapy
Industry
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Star Therapeutics Presents Data on Investigational VWD Therapy
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BioMarin to Narrow Its Commercial Focus for Hemophilia A Gene Therapy
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Gene Therapy a pill with DNA
Pfizer Announces New Data from Hemophilia A Gene Therapy Study
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FDA Approval Includes Full ALTUVIIIO™ Pediatric Trial Results
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Gene Therapy
Hemophilia B Gene Therapy Trial Data Published
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Takeda Announces Interim Trial Results for Investigational ITP Therapy
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uniQure Announces Findings from HCC Case Investigation in HOPE-B Trial for Gene Therapy
Takeda's Voluntary Withdrawal: Alert on Misprinted Labels for VONVENDI®
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FDA Accepts Biologics License Application for Marstacimab
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CSL Presents Three Year Treatment Data for Hemophilia B Gene Therapy
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FDA Grants Expanded Approval to Wilate® for Prophylaxis in All Types of…
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First Patient Dosed with Hemophilia Gene Therapy Candidate ASC618
Industry
Sernova Receives Dual FDA Designations for Investigational Hemophilia…