The Italian breakthrough in CRISPR trials for rare diseases: a focus on beta-thalassemia and sickle cell disease treatment



type one von willebrand's disease :: Article Creator

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pharmaphorum6y Shire's von Willebrand disease therapy Veyvondi approved in EU

These include a phase 1 multicentre, controlled, randomised, single-blind, dose-escalation study of the safety, tolerability and pharmacokinectics in subjects 18 to 60 years of age with severe VWD.






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