Wolfgang Miesbach: WFH’s First AAV Gene Therapy Chapter Marks a New Era in Hemophilia Care
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
”Treatment for ALL: Diagnosis is rising — but the gap remains vast.
Haemophilia diagnoses climbed to approximately 290,000 (from around 245,000 in 2020) and VWD to around 115,000.
Yet two-thirds of people with haemophilia, and most with VWD (approximately 1 in 1,000), are still undiagnosed.
Humanitarian aid scaled past its target. 34,747 people have now been treated through the programme, including 4,000 children reaching prophylaxis.
The registry became usable.
Past its 10,000-registration target, the new WBDR Data Dashboard lets HTCs and researchers actually interrogate the data — by type, severity, age, and region.
The WFH published its first AAV gene therapy guideline chapter, alongside a shift to a Living Guidelines Model that updates as evidence emerges.
For a field moving this fast, that shift may matter as much as the recommendations themselves.
Behind every number is the WFH‘s core mission: working with NMOs, treatment centres, and healthcare providers worldwide to bring diagnosis and care to everyone with an inherited bleeding disorder — Treatment for ALL.”
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