Catherine Rea, Thomas York, Ulrike Lorch, Kate Khair, Luke Pembroke, Jigar Amin

New insights from a prospective study on Glanzmann thrombasthenia (GT) have been published in Blood (2023). Led by Catherine Rea, Thomas York, Ulrike Lorch, and their team, this observational study delves into the bleeding patterns and impacts on quality of life (QOL) in 30 GT patients over 3 months. Using a custom-designed electronic bleed diary, the study reveals the high frequency of bleeds and their substantial socioeconomic impacts. Results detail bleed rates, anatomical locations, associated symptoms, and the burden on daily activities. The findings underscore the urgent need for improved treatments to reduce bleed frequency and enhance QOL for GT patients. This study establishes a direct correlation between bleed events and their profound effects on patients' lives, advocating for advancements in GT care.

Latest news

Five Years Since a World-First CRISPR Breakthrough at Richmond Pharmacology

December 8, 2025
In November 2020, Richmond Pharmacology made medical history when a patient received the first-ever systemically delivered CRISPR therapy, using lipid nanoparticles to edit genes directly inside the human body.
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Events

JSCPT 2025

5 – 6 December 2025
Richmond Pharmacology will attend JSCPT 2025 in Tokyo, represented by their leadership team. Topics include early-phase studies, bridging studies between Europe and Asia, gene and RNA-based therapy development, and cross-border collaboration.
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