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Emerging Trends in Rare Disease Trials: Orphan Drugs, Genetics & Patient Voices

Rare disease trials in 2024-2025 are reshaped by genetic therapies, refined orphan drug policies, and empowered patient voices, offering new hope and access pathways for ultra-rare conditions.

Robert Maxwell Aug 13, 2025

orphan drug access ultra-rare genetic diseases rare disease support gene therapy research

How to Navigate Rare Disease Treatments & Access Orphan Drug Trials

Navigating rare disease treatments and accessing orphan drug trials can be challenging. Discover five key steps—from understanding your rights to finding clinical trial opportunities—that empower patients with uncommon genetic disorders.

Robert Maxwell Aug 11, 2025

orphan drug access ultra-rare genetic diseases rare disease treatments patient-centered research