Explore our latest articles, insights, and stories from the frontlines of clinical trials.
A practical Q&A on the future of orphan trials: how to find studies for rare pediatric diseases, what to expect from gene therapy, compassionate use basics, and where to get financial and travel help.
A practical, patient-first guide to matching rare disease trials: step-by-step matching, travel and lodging help, assessing risks vs benefits, and creating school plans for children with rare conditions.
A mother's story of navigating compassionate-use access to an orphan drug, building a back-to-school flu plan for her child with a rare disease, and finding travel and financial aid for trial participation.
Discover data-driven insights on navigating treatment options for orphan diseases, timeline strategies, patient stories, and how biotech founders and clinical research advance rare condition therapies.
For families navigating rare disease trials, technology advances are transforming hope into action. Discover how digital tools, patient stories, and regulatory progress are reshaping access and treatments for ultra-rare conditions.
Living with ultra-rare disorders demands navigating complex treatment paths. Through real patient stories from rare autoimmune trials and compassionate use insights, this post explores how emerging platforms and regulatory expertise are reshaping access to care.
Exploring how families navigate rare disease clinical trials reveals critical insights into gene therapies, orphan drug access, and patient-centered support that drive meaningful outcomes in ultra-rare pediatric conditions.
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.
Explore data-driven insights into navigating treatment options for rare diseases, understanding orphan drug benefits, safely participating in gene therapy trials, and managing life with ultra-rare conditions amid evolving regulatory trends.