From Bleeding to Swelling – 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
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