Blog

Future of Orphan Trials: Gene Therapy, Support & Compassionate Use

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.

Case Study: Families Finding Gene Therapy Trials via Telehealth

Families like Mara's and seniors like Leonard are using telehealth, registries, and decentralized research to find gene therapy trials, navigate expanded access, and prepare for treatment journeys.

Step-by-Step: Compassionate Use, Trial Aid & Back-to-School Flu Plan

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.

How Clinical Trials Transformed Patient Outcomes in Rare Disease Cases

Discover how clinical trials are transforming rare disease treatment with patient success stories and actionable steps to navigate emerging gene therapies and improve outcomes.

How to Navigate Rare Disease Trials & Harness AI for Breakthroughs

Navigating rare disease trials demands innovative strategies. AI-driven diagnostics and adaptive trial designs are transforming treatment breakthroughs, improving cost-effectiveness, and empowering patients in ultra-rare condition research.

The Future of Rare Disease Care: Gene Therapy and Trials Near You in 2024

2024 marks a pivotal shift in rare disease care, with gene therapy advancing rapidly. This analysis explores recent trials, patient stories, and emerging trends that redefine treatment options and clinical trial access for orphan conditions.

Navigating Rare Disease Trials: Expert Insights on Gene Therapies & Patient Stories

Explore a practical guide to navigating rare disease treatment options with expert insights on gene therapies, orphan drug clinical trials, and inspiring patient stories in ultra-rare conditions.

Navigating Rare Disease Trials: Real Family Success with Gene Therapies & Patient Support

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.

Navigate Rare Disease Care: Practical Tips on Orphan Drugs & Gene Trials

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.