Meeting regarding the rare genetic disease of Diamond Blackfan Anemia Syndrome with patients, caregivers, FDA, medical product developers, healthcare providers and federal partners to give voice to the debilitating nature of DBA and the urgent need for a cure.
DARIEN, CT, December 8, 2025 — Styrke Foundation is pleased to announce it will host an Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting focused on Diamond-Blackfan Anemia Syndrome (DBAS) as a parallel effort to FDA’s PFDD initiative to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions. The meeting will be held virtually on Friday, May 1, 2026, beginning at 11 a.m. ET, and is open to all members of the DBAS community, including patients, caregivers, clinicians and researchers. This meeting will give voice to the profound burden of this rare genetic hematological disorder and the urgent need for meaningful treatment development.
EL-PFDD meetings give FDA and other key stakeholders, including medical product developers, health care providers, federal partners, an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. Patient input may inform FDA’s decisions during drug development and application review.
DBAS is a severe congenital hematological bone marrow failure syndrome typically diagnosed in infancy or early childhood. The condition is characterized by the body’s inability to produce sufficient red blood cells, leading to chronic anemia, lifelong transfusion dependence, iron overload, developmental challenges, and significant medical fragility. At present, the only known hematological curative option is a full stem cell (bone marrow) transplant – an extremely costly, high-risk treatment that requires a suitable donor match, which many patients never find. Beyond this option, no cure or disease-modifying therapy exists.
“The challenges faced by DBAS patients and families are immense and far too often overlooked,” said Anne Yang, Executive Director of Styrke Foundation. “This PFDD meeting represents a vital moment for the DBAS community to speak directly to regulators and decision-makers about the lack of effective treatments and the urgent need for safer, more viable long-term therapies.”
Quote from the father of a teenage boy living with DBAS:
“Children and families who live with DBA, live under the fear of what comes next. They long for moments free of the burden of constant suffering, mental anguish and pain exacerbated by the incessant cadence of hospitalizations. Parents do their best to fabricate fleeting moments of normalcy that end up being little more than a mirage of hope. All the while, they live with the knowledge that their child’s life will come to an early end after suffering through many of the most painful and damaging treatments modern medicine has to offer. FDA’s willingness to see our tears and hear our cries for help is one of the greatest gifts we could hope for.”
Participants will share their personal experiences through live testimony, polling, and written comments. Styrke Foundation is working in collaboration with Styrke Foundation is working in collaboration with DBAS patient advocacy organizations globally to ensure broad participation and a full representation of the patient and caregiver experience.
Event Details
Title: DBAS EL-PFDD Meeting
Date: Friday May 1, 2026
Time: 11 a.m. ET
Location: Virtual meeting link will be available on www.styrke.org
Contact
Anne Yang
Executive Director
Styrke Foundation for Rare Disease Research and Treatment
(917) 797-0233
ayang@styrke.org
About Styrke Foundation
Styrke Foundation’s 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, visit www.styrke.org.