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Conference Briefings

Relationship Between Transgene-Produced FVIII and Bleeding Rates 2 Years After Gene Transfer With Valoctocogene Roxaparvovec: Results From Gener8-1

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

Stable Hemostatic Correction and Improved Hemophilia-Related Quality of Life: Final Analysis from the Pivotal Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec

Steven W. Pipe, MD

Elucidating the Mechanism Behind the AAV-Derived Factor VIII Assay Discrepancy

 Anna Sternberg, PhD

Delivery of CRISPR/Cas9 mRNA LNPs to Repair a Small Deletion in FVIII Gene in Hemophilia A Mice

Chun-Yu Chen, PhD

Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients With Hemophilia B

Guy Young, MD

Stabilin-2 Promoter Modulates the Immune Response to FVIII After Lentiviral Vector Delivery in Hemophilic Mice

Antonia Follenzi, MD, PhD

Improvements in Health-Related Quality of Life in Adults With Severe or Moderately Severe Hemophilia B After Receiving Etranacogene Dezaparvovec Gene Therapy

Steven W. Pipe, MD

A Factor VIII Variant With Elimination of the N-glycosylation Site at 2118 Reduced Anti-FVIII Immune Responses in Gene Therapy Treated Hemophilia A Mice

Carol H. Miao, MD

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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

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