Patient Focused Drug Development
Apriligen is committed to ensuring that the voices of DBAS patients and their families are at the center of drug development, working alongside the Styrke Foundation to bring real-world patient experiences directly to the FDA and the broader research community.
The Styrke Foundation
Apriligen is proud to be a grant-funded partner of The Styrke Foundation for Rare Disease Research and Treatment, a 501(c)(3) nonprofit organization dedicated to funding cutting-edge research into rare and ultra-rare genetic pediatric diseases.
Founded on a legacy of courage and resilience by a family whose loved one lives with an ultra-rare condition, Styrke is committed to bridging the gap between scientific discovery and clinical application.
The Foundation's work directly supports programs like APR-2020 that are advancing toward clinical trials, with the goal of developing life-changing therapies that can be scaled using platform technology to benefit the thousands of children worldwide living with conditions for which there is currently no cure.
Externally Led Patient-Focused Drug Development (EL-PFDD) Meeting
The Styrke Foundation hosted an Externally Led Patient-Focused Drug Development (EL-PFDD) meeting dedicated to Diamond-Blackfan Anemia Syndrome (DBAS) on May 1…The Styrke Foundation hosted an Externally Led Patient-Focused Drug Development (EL-PFDD) meeting dedicated to Diamond-Blackfan Anemia Syndrome (DBAS) on May 1, 2026 at 11:00 AM EST.
This meeting provided the U.S. Food and Drug Administration (FDA), medical product developers, clinicians, and academic researchers with a direct opportunity to hear from individuals living with DBAS about the health effects and daily impacts of their condition, their treatment goals, and the decision factors they consider when seeking or selecting a treatment.
Conducted as a parallel effort to the FDA's own PFDD initiative, the meeting covered two key topics: the symptoms and daily burden of DBAS, and current approaches to treatment including what patients and caregivers would look for in an ideal therapy.
Patient input gathered through this meeting can support FDA benefit-risk assessments, advise drug sponsors on their development programs, identify areas of unmet need, and raise awareness and engagement within the DBAS patient community.
Watch the EL-PFDD Meeting
DBAS Patient and Caregiver Survey
In preparation for the EL-PFDD meeting, the Styrke Foundation launched a comprehensive DBAS Patient and Caregiver Experience Survey designed to capture the real-world perspectives of individuals and families affected by Diamond-Blackfan Anemia Syndrome.
Facilitated through grant funding from the Styrke Foundation, we believe it will be one of the most thorough, real-world assessments ever conducted within the DBAS community.
The survey spans nine sections covering patient demographics, health effects and daily impacts, medical journey, current treatment approaches, emotional and social impacts, caregiver experience, financial burden, and future outlook.
Responses are anonymous and voluntary, and the data collected will be shared with the FDA as part of the PFDD initiative, directly informing regulatory decision-making and the development of new therapies.
We strongly encourage all DBAS patients, caregivers, and family members to participate and make their voices heard.
