Therapeutic Genome Editing – Reshaping the Landscape of Advanced Therapies
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Our network spans the globe, just like your patients do
Patient advocacy group relationships and social media outreach help reach into affected communities.
Meanwhile, our established relationships with key opinion leaders and clinical research sites around the globe secure access to a wide range of patient populations.
A sponsor selected Precision to manage a global natural history study in a rare, X-linked kidney disorder. The study had been recommended by the FDA because there was extremely limited published data on this rare disease—the goal was to characterize renal function and decline in patients with the disease prior to initiating a phase 2/3 clinical study. The study, to be conducted in the United States, Canada, Australia and in Europe, would follow 200+ patients for approximately 2 years.
Enrollment was by far the greatest study challenge, combining a low disease prevalence and a primary target population—young males—with life commitments such as school and work that left little time for participation in a trial that offered no therapeutic benefit for participants and would require traveling to a clinic every 3 to 6 months.
Discover expert analysis and broaden your understanding through Precision’s Insights on current research, case studies, and trends.