Recently Approved Cell-Based Gene Therapies Demonstrate Innovation in Sickle Cell Disease Treatment
This white paper offers a comprehensive primer on the burden of SCD, the patient journey, and current and emerging treatment options.
Resource Guide for Patients Living with Rare Diseases
Download our Resource Guide for Patients Living with Rare Diseases to find essential information and support to understand your rare disease diagnosis, connect with advocacy organizations, and explore treatment options.
Integrated REMS and Patient Support Services Solution for Complex Rare Disease
Discover how UBC has been the single point of contact for over 10 years to reduce program risk and burden through multiservice governance for 28,000 patients.
First-in-Class Therapy Succeeds with Personalized Patient Services
This reimbursement hub for rare disease patients quickly expanded to cover adherence, resulting in great patient satisfaction and results.
Optimizing Evidence Generation in Rare Disease Studies with Patient-Centric Strategies
RWD and RWE are emerging as strategic tools in rare disease study design to address research challenges with real-world solutions.
