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

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Navigating Rare Disease Treatments: Insights from Orphan Trials & Gene Therapy Access

Navigating rare disease treatments involves understanding orphan trials, gene therapy access, and managing symptoms with emerging drugs—balancing innovation with cost-effectiveness amid evolving FDA and EMA guidance.

Robert Maxwell Jul 29, 2025

rare disease treatments orphan drug research gene therapy trials ultra-rare conditions

How Gene Therapies & Orphan Drug Access Are Shaping Rare Disease Care

Discover how gene therapies and orphan drug access are revolutionizing rare disease care. Learn about navigating treatment options, patient stories, and the role of technology and coordinators in transforming outcomes.

Robert Maxwell Jul 24, 2025

orphan drug access ultra-rare disorders gene therapy trials rare disease treatments