Emerging Trends in Patient-Centered Trials and Gene Therapy for Rare Diseases
Explore how patient-centered orphan drug trials and cutting-edge gene therapy are transforming rare disease treatment. This deep dive covers global regulations, patient rights, and the critical role of innovative clinical trial platforms in advancing care for ultra-rare disorders.
Robert Maxwell
Sep 03, 2025
orphan drug research
rare genetic disorders
patient-focused trials
gene therapy studies