Advancing a Coordinated Rare Disease Ecosystem
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Rare Disease Day is observed each year on the last day of February to raise awareness for conditions that affect a small percentage of the population, yet collectively impact 350 million people worldwide, including more than 30 million in the United States, many of them children.
BioNTX is proud to support this community by fostering awareness, promoting knowledge, and standing alongside clinicians, researchers, patients, families, and caregivers in their journey.
Rare Disease Day is a moment to reflect—and more importantly, it is a call to act.
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A BioNTX Commitment to Advancing Rare Disease Innovation
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(left) Kasey Woleben, Co-Founder of Rare Village Foundation & Cure Mito Foundation
(right) Casey McPherson, Founder & CEO of To Cure a Rose Foundation & AlphaRose Therapeutics
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Across research, clinical care, and therapeutic development, rare diseases reveal the pressure points of our healthcare and innovation systems. Fragmented data. Lengthy diagnostic journeys. Regulatory pathways not designed for today’s science. Investment models that struggle to keep pace with complexity. And patients and families who cannot live with delays.
At BioNTX, we believe addressing rare disease challenges requires more than isolated solutions. It requires coordination across the full ecosystem—researchers, clinicians, biotech innovators, health systems, funders, policymakers, and enabling technologies—working with shared urgency and aligned intent.
BioNTX is committed to serving as a convener, connector, and catalyst—bringing together diverse efforts, amplifying proven models, and helping translate innovation into real-world impact for rare disease communities.
This work is ongoing. The stakes are high. And the time to move is now. Please reach out if you are interested in this topic.
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John Crowley of BIO Connects with North Texas
at the 2025 BioNTX Summit
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At the 2025 iC³ Life Science & Healthcare Innovation Summit, John Crowley, Chairman & CEO of BIO, joined leaders from across the life sciences ecosystem at the Patient Advocacy Luncheon. The convening brought together industry executives, innovators, and organizations committed to advancing rare disease research and improving pathways from discovery to care.
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Texas Rare Disease Policy Snapshot:
Progress, Gaps, and Opportunities
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The National Organization for Rare Disorders (NORD) developed the State Report Card® to evaluate all 50 states and Washington, DC across nine critical policy areas affecting the 30 million Americans living with rare diseases and their families. Designed as a practical tool for patients, advocates, and policymakers, the State Report Card® helps guide efforts to strengthen state policies and improve support for the rare disease community.
Texas shows mixed performance in the NORD State Report Card®, demonstrating moderate progress in several areas while still facing significant policy gaps. The state earns solid marks for newborn screening, telehealth access, step therapy protections, and prescription drug affordability, while eligibility support for pregnant women and children falls in the mid-range. However, limited Medicaid eligibility, gaps in insurance protections, and the absence of a Rare Disease Advisory Council continue to create barriers to care access and financial protection for Texans living with rare diseases and their families.
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BioNTX encourages you to raise awareness for Rare Disease Day and show your support for the rare disease community.
We invite you to use your social media platforms through stories or posts to highlight the day and encourage your followers to Show Your Stripes in support of those affected.
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