The University of Texas at Arlington is creating a new kind of graduate program that trains doctoral students to become experts in a single rare disease while moving their research from the lab to real-world diagnosis, clinical trials and treatment.
The initiative builds on UT Arlington's collaboration with Cook Children's Health Care System. Last March, UTA President Jennifer Cowley welcomed representatives from 16 family foundations to campus for the Rare Disease Family Foundations Symposium. During the event, families shared their origin stories and described the challenges they face, particularly the "diagnostic odyssey," during which patients often spend years seeking an accurate genetic diagnosis. "Fragmented" was a common term used to describe the barriers patients encounter on their path, not only in diagnosis, but across data, care, research and advocacy.
Dennis Lal, a UTA professor and executive director of the Center for Innovation in Health Informatics (CIHI), as well as an executive scientist in Cook Children's Pediatric Precision Health Program, is leading the initiative and developed the innovative approach to training Ph.D. candidates.
"Our vision is to train a new generation of translational leaders—genetic counselors, scientists and physicians—who do not merely study rare diseases, but become domain experts in one disorder while being fluent across the full rare disease ecosystem, from patient priorities and clinical care to diagnostics, data and therapeutic development," Dr. Lal said.
"This is not a traditional Ph.D. program with a disease as a case study. It is a disease-anchored doctorate cocreated with patient organizations and designed to connect lived experience, specialty care, research and industry in ways that accelerate cures, elevate patient voices and reshape how rare diseases are studied, funded and treated."
Related: UTA appoints Lal to lead precision health, informatics
Under the new curriculum, each student's coursework, clinical exposure, research rotations and dissertation will be tailored to a single disease. The program aligns discovery, clinical translation, lived experience and therapeutic development around real patients, with training opportunities spanning patient organizations, Cook Children's specialty clinics, research laboratories and industry partners.
One goal of the symposium was to pair family foundations with the program, inviting each to sponsor a disease-anchored Ph.D. student. The FamilieSCN2A Foundation has already committed to supporting one.
"Our commitment to sponsoring a disease-anchored Ph.D. student is about ensuring SCN2A stays front and center in both discovery and clinical translation," said Leah Schust Myers, founder and executive director of FamilieSCN2A. "By embedding dedicated expertise within the Cook–UTA ecosystem, we are investing in sustained progress—building the knowledge, focus and continuity this field needs to move faster for families."
The Ph.D. track in quantitative biology will be housed in the College of Science.
The initiative represents the latest example of UTA strengthening its innovative research and graduate education. Additionally, UTA could become a potential convening platform for patient organizations, biotech, and clinical leaders seeking a scalable rare disease ecosystem. This approach resonated with the foundations that attended the symposium.
"This event reinforced the importance of building coordinated ecosystems across research, clinical care and training," said Gabrielle Rushing, chief scientific officer at the CSNK2A1 Foundation. "Disease-anchored Ph.D. programs are a key part of that, developing scientists who can translate discovery more efficiently into patient-centered outcomes. Collaboration like this is what will move rare disease treatment forward."
Ilene Penn Miller, executive director of the Rare Epilepsy Network, said UTA and Cook Children's are answering an urgent call to build a precision-ready ecosystem that connects patient communities to the clinic, lab and industry.
"When we learn from every patient, families get earlier answers, better coordinated care, and real access to life-changing therapies," Miller said.
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For President Cowley, the initiative reflects the kind of transformative work she wants to see across UTA's campus.
"I see people united by a shared commitment to developing treatments and cures for rare diseases, and that mission aligns perfectly with who we are as a comprehensive research university," Dr. Cowley said.
About The University of Texas at Arlington (UTA)
The University of Texas at Arlington is a growing public research university in the heart of Dallas-Fort Worth. With a student body of over 42,700, UTA is the second-largest institution in the University of Texas System, offering more than 180 undergraduate and graduate degree programs. Recognized as a Carnegie R-1 university, UTA stands among the nation's top 5% of institutions for research activity. UTA and its 280,000 alumni generate an annual economic impact of $28.8 billion for the state. The University has received the Innovation and Economic Prosperity designation from the Association of Public and Land Grant Universities and has earned recognition for its focus on student access and success, considered key drivers to economic growth and social progress for North Texas and beyond.
