Haemophilia and Hereditary Angioedema – A Shared Therapeutic Revolution in Rare Diseases – RPTH Journal
RPTH Journal shared a post on LinkedIn about a recent article by Massimo Cugno et al., adding:
“Haemophilia and hereditary angioedema: two rare diseases, one shared therapeutic revolution
Haemophilia and hereditary angioedema (HAE) are both rare monogenic disorders involving dysregulation of serine protease pathways, blood coagulation and the kallikrein–kinin system, respectively.
Their clinical manifestations could not be more different: bleeding vs angioedema.
Yet their therapeutic journeys have evolved in strikingly parallel ways.
A new review Massimo Cugno and Pier Mannuccio Mannucci in RPTH Journal traces this parallel evolution, from plasma-derived products in the 1970s to the cutting edge of gene and RNA-based therapies today.
The therapeutic trajectories of haemophilia and HAE illustrate a broader principle: deep mechanistic understanding of rare diseases can drive the transition from managing acute episodes to achieving long-term disease control, and ultimately, cures.”
Title: Hemophilia and hereditary angioedema: parallel therapeutic advances in genetic diseases of serine protease pathways
Authors: Massimo Cugno, Pier Mannuccio Mannucci
Stay updated on all scientific advances with Hemostasis Today.
-
Jul 23, 2026, 22:20Thrombus NETs Predict Long-Term Ischemic Stroke Recurrence – JTH
-
Jul 23, 2026, 21:16Applications Still Open for the 13th ESO Edinburgh Stroke Research Workshop – European Stroke Organisation
-
Jul 23, 2026, 20:46Grace Choung: A Novel CFHR5 Copy Number Variant in Postpartum Atypical HUS
-
Jul 23, 2026, 20:10Mohammad Alzoubi: One Month of Dual Antithrombotic Therapy After PCI Was Non-Inferior to 12 Months
-
Jul 23, 2026, 20:08Ahmed Bennis: Calcitriol Replenishment May Mitigate Ischemic Risk in MASLD
-
Jul 23, 2026, 20:06Mitsuaki Sawano: Can Cardiovascular Risk Prediction Models Developed in One Population be Reliably Applied to Another?
-
Jul 23, 2026, 20:05Abdul Muqtadir Abbasi: Hemostasis Isn’t About Memorizing Factor Numbers – It’s About Recognizing Patterns
-
Jul 23, 2026, 19:40Maureen Macharia: How Apixaban Transformed Modern Anticoagulation
-
Jul 23, 2026, 19:30Jordan Agay: Why We Need to Track the Neurological Costs of Anemia