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Explore our latest articles, insights, and stories from the frontlines of clinical trials.

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How Gene Therapy Transformed Ultra-Rare Disease Outcomes: Real Patient Stories

Discover how gene therapy is revolutionizing outcomes for ultra-rare diseases through personalized treatment and real patient stories. Learn actionable steps to navigate clinical trials and prepare for your first specialist visit.

Robert Maxwell Jul 14, 2025

orphan drug research ultra-rare diseases gene therapy trials rare condition treatments

Navigating Rare Disease Treatments: How to Access Cutting-Edge Gene Therapy Trials

Navigating treatment options for rare diseases can be daunting. This blog unpacks how gene therapies are transforming care, shares caregiver insights, and offers tips on accessing cutting-edge clinical trials for ultra-rare conditions.

Robert Maxwell Jul 03, 2025

orphan drug access ultra-rare disease care gene therapy research rare condition treatments