Clinical Trial Efficacy and Safety

Novel Approaches Under Investigation

Monthly Roundup: May 2024

Monthly Roundup: April 2024

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

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

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

Efficacy and Safety of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A: Results From the GENEr8-1 Two-Year Analysis

Final Analysis From the Pivotal Phase 3 HOPE-B Gene Therapy Trial: Stable Steady-state Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderately Severe Hemophilia B

Dose Selection and Study Design for B-LIEVE, A Phase 1/2 Dose Confirmation Clinical Trial of FLT180A Gene Therapy for Patients with Hemophilia B

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated With Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Factor IX Expression Within the Normal Range Prevents Spontaneous Bleeds Requiring Treatment Following FLT180a Gene Therapy in Patients With Severe Hemophilia B: Long-term Follow-up Study of the B-Amaze Program

Current Update of Clinical trials on Gene Therapy for Hemophilia

Monthly Roundup

Interactive Webinars

Podcasts

Conference Coverage

Highlights From the 17th Annual Congress of EAHAD

Highlights From the 65th ASH Annual Meeting and Exposition

Highlights From the ESGCT 30th Annual Congress

Highlights From the 31st Congress of ISTH

Highlights From the 32nd Congress of ISTH

Gene Therapy Illustrated Clinical Trials

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Supported through educational grants from BioMarin, CSL Behring, Pfizer Inc., Roche, and Spark Therapeutics

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