Launched today is a global survey of the DBAS Patient and Caregiver Experience to support leading medical research in the rare genetic disease of Diamond-Blackfan Anemia.
DARIEN, CT, January 28, 2026 — Styrke Foundation is pleased to announce the launch of the DBAS Patient & Caregiver Experience Survey. Responses will provide clinicians, patients, patient advocates, medical researchers and other interested parties with a personalized and unfiltered understanding of the critical need to find a cure for DBAS.Survey results will support research, expedite the development of new and better treatments, and inform regulatory decision-making.
Portions of this survey may also be shared with the U.S. Food & Drug Administration (FDA) as part of the Patient-Focused Drug Development (PFDD) Initiative to be shared during the EL-PFDD meeting organized by the Styrke Foundation on May 1, 2026 at 11am EST.
All individual survey responses will be confidential and all data and analysis will be anonymized.
“Once completed, we believe this will be one of the most comprehensive, real-world assessments from the DBAS community to improve the treatment landscape in this debilitating disease” said Anne Yang, Executive Director of the Styrke Foundation.
“This survey, along with the upcoming PFDD meeting with Styrke Foundation, FDA, patients and members of the medical community represents a highly visible platform for the DBAS community to have their voices heard and impart the urgency for viable long-term treatments. Our mission is to bring cures to patients and their families – by making their voices heard in a way that leverages the power of their community. We are confident that all stakeholders will be moved to action.”
From the mother of a 7-year-old daughter with DBAS,:
For years, it felt like no one was really listening. Now we finally have a chance to show the FDA and researchers what life with DBA is actually like – the daily reality, not just the medical textbook version. It means a lot to know other families are doing this alongside us. For the first time, it feels like something might actually change.
DBAS is a severe hematological bone marrow failure syndrome diagnosed in infancy or early childhood. The condition is characterized by the body’s inability to produce red blood cells, leading to chronic and complete anemia, lifelong transfusion dependence, iron overload, developmental challenges, and significant medical fragility.
Without regular medical intervention and treatment, DBAS is terminal. At present, the only curative option is a stem cell (bone marrow) transplant – an extremely costly, high-risk treatment requiring myeloablative chemotherapy and a suitable donor match, which many patients never find. Beyond this option, no cure or disease-modifying therapy exists.
To participate in the survey please go to www.styrke.org. The estimated time required to complete the survey is 20 to 30 minutes. For more information on the PFDD meeting and to participate as a patient, patient advocate, clinician or other interested party, please register your interest at https://styrke.org/contact/ or send an email to info@styrke.org.
Contact
Anne Yang
Executive Director
Styrke Foundation for Rare Disease Research and Treatment
+00 1 (917) 797-0233
ayang@styrke.org
www.styrke.org
About the Styrke Foundation
The Styrke Foundation for Rare Disease Research and Treatment is a non-profit registered as a 501(c)(3) in the US. Our mission is to create a world where children affected by rare genetic diseases can live fulfilling lives without limitations.
Through advocacy, patient support, research advancement, and community engagement, Styrke works to empower families and accelerate progress toward safe, effective, and accessible treatments for children worldwide.
For more information, visitwww.styrke.org. To support our mission and bring cures to diseases like DBAS, please consider making a tax-deductible donation here https://styrke.org/support/donate/.