Histopathological characterization of immune-mediated hepatocellular injury following gene therapy in hemophilia

Conference Coverage

Durable efficacy and safety in responders: 5-Year post hoc end-of-study analysis of etranacogene dezaparvovec in the phase 3 HOPE-B trial

Conference Coverage

Improved hFIX and hFVIII expression with ITR-modified AAV vectors in hemophilic mice

Conference Coverage

Evolution Directed Gene Engineered (EDGE) Gain-of-Function Factor VIII Variants Enhance Gene Therapy for Hemophilia A

Conference Coverage

Use of Fidanacogene Elaparvovec, a Gene Therapy Vector, to Deliver a Stable, Fully Functional Human Factor IX Transgene for the Treatment of Hemophilia B: A Combined Analysis of Safety

Conference Coverage

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults With Moderately Severe to Severe Hemophilia Α

Conference Coverage

BeCoMe–9: A Phase 1/2 Dose Escalation and Expansion Study of be-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

Conference Coverage

Monthly Roundup: December 2024

Monthly Roundup

Monthly Roundup: November 2024

Monthly Roundup

Monthly Roundup: October 2024

Monthly Roundup

Gene Therapy in Hemophilia – Novel and Emerging Therapies

Interactive Webinars

Gene Therapy in Hemophilia: Clinical Care Strategies for Gene Therapy

Interactive Webinars

Monthly Roundup: September 2024

Monthly Roundup

Monthly Roundup: August 2024

Monthly Roundup

Efficacy and Safety of Fidanacogene Elaparvovec in Adults with Moderately Severe or Severe Hemophilia B: Updated Results From the Phase 3 BENEGENE-2 Gene Therapy Trial

Highlights From the 32nd Congress of ISTH

Monthly Roundup: July 2024

Monthly Roundup

Health-related Quality-of-Life Outcomes 4 Years After Treatment with Valoctocogene Roxaparvovec

Highlights From the 32nd Congress of ISTH

Efficacy and Safety of Valoctocogene Roxaparvovec 4 Years After Gene Transfer in GENEr8-1

Highlights From the 32nd Congress of ISTH

Monthly Roundup: June 2024

Monthly Roundup

Novel Approaches Under Investigation

Microlearning

Neutralizing Antibodies

Microlearning

Durability of Gene Therapy

Microlearning

Monthly Roundup: May 2024

Monthly Roundup

Stable and durable factor IX (FIX) levels over 4 years after etranacogene dezaparvovec gene therapy administration in a Phase 2b trial in patients with haemophilia B

Highlights From the 17th Annual Congress of EAHAD

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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

Microlearning

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

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