Wolfgang Miesbach: How to Better Understand and Explain Gene Therapy of Haemophilia?
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on Linkedin:
“How to better understand and explain gene therapy of haemophilia? Our latest publication, ‘Gene-based therapies for hemophilia,’ is now available as full-open access article in Research and Practice in Thrombosis and Haemostasis! This illustrated review provides a comprehensive overview of gene therapy for hemophilia A and B, highlighting how recent advances are transforming patient care.
Through 10 detailed figures, the article breaks down complex concepts into clear visuals, making it easier for both patients and healthcare professionals (HCPs) to understand:
· Mechanisms of gene therapy: How adeno-associated virus (AAV) vectors deliver functional FVIII or FIX genes to address the root cause of hemophilia
· Clinical outcomes: Visual summaries of trial results for therapies like valoctocogene roxaparvovec, etranacogene dezaparvovec, and fidanacogene elaparvovec, showing sustained factor production and reduced bleeding episodes
· Benefits for patients: Figures illustrate the potential for long-term freedom from regular infusions and improved quality of life
· Challenges and future directions: Visuals explain ongoing issues such as immune responses, hepatotoxicity, and strategies for expanding eligibility and durability
These figures are invaluable for:
· Educating patients about what gene therapy means for their treatment journey
· Empowering HCPs with up-to-date, evidence-based visuals to support informed discussions and shared decision-making
Read the full open-access article to explore all 10 figures and deepen your understanding of gene therapy’s promise for haemophilia patients.”
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