A landmark step in FAST’s strategy to remove barriers, expand clinical trial capacity, and shape what’s possible in rare disease research.
On August 14, 2025, FAST and Rush University Medical Center opened the doors to the Rush Pediatric Neurosciences F.A.S.T. Center for Translational Research, a groundbreaking clinical hub built to serve families, train clinical researchers, and accelerate the development of new treatments for individuals living with Angelman syndrome. Housed within Rush’s nationally recognized pediatric neuroscience program, the Center provides a dedicated environment for advancing therapies, supporting families through clinical trials, and training the next generation of rare disease specialists. Established with a $5 million gift from FAST in December 2022, it is led by longtime FAST partner and renowned clinician-scientist Dr. Elizabeth Berry-Kravis.
Designed for Urgency
The Center was developed to address a critical gap in rare disease research: most hospital systems lack the trained personnel, trial facilities, and bandwidth to manage the growing pipeline of Angelman syndrome clinical trials now moving into early-stage testing.
“We’re at a point in the scientific journey where the limiting factor isn’t ideas, it’s execution,” said Dr. Allyson Berent, chief science officer at FAST. “This Center was designed to close that gap.”
With dedicated staff, coordinated trial operations, and purpose-built facilities, the Center can pivot quickly when a therapy is ready for testing, reducing months, or even years, of typical delays. At the same time, its training programs ensure a growing pool of clinicians and researchers are ready to run these complex studies in the future.
A Platform for Progress in Rare Disease Clinical Trials
The Center combines hands-on training with the resources needed to support the full spectrum of translational research, with priorities including:
Advancing novel therapeutics with the potential to address the root cause of Angelman syndrome
Coordinating natural history studies, biomarker development, and other critical tools that make trials possible and reduce risk
Supporting clinical research that fills knowledge gaps and drives the development of new therapies
Its training program for rare neurodevelopmental conditions is the first-of-its-kind, preparing physicians to lead complex trials at their home institutions through externships and immersive instruction in:
Regulatory navigation, ethics, and contracting
Facility setup for trial-related diagnostics and care
Building and leading cross-functional trial teams (neurology, anesthesia, radiology, neurosurgery, neuropsychology, etc.)
Advanced drug delivery methods for novel therapeutics
While the Center’s early work is focused on Angelman syndrome, its model is designed to help other rare neurodevelopmental disorder trials overcome similar operational challenges, from early-phase testing through larger-scale studies.
What It Means for Families and the Field
The opening of the F.A.S.T. Center marks a turning point for the Angelman syndrome community. It will:
Accelerate access to promising new treatments
Improve readiness for first-in-human trials
Expand the clinical workforce equipped to deliver advanced therapies
Serve as a model for advancing therapies safely and efficiently
“For families, time matters,” said Dr. Elizabeth Berry-Kravis, director of the Rush Pediatric Neurosciences F.A.S.T. Center. “Every part of this Center is designed with patients and families at the heart, so that when hope turns into a real possibility, we’re ready to deliver it. This is the next era in delivering life-changing treatments.” That readiness turns hope into measurable progress. For families, it means that when a promising therapy emerges, there is a clear path to testing, not years of waiting for the right systems and staff to be assembled.
Shaping the Future of Rare Disease Research
FAST is redefining what’s possible in rare disease science and the Rush Pediatric Neurosciences F.A.S.T. Center for Translational Research is at the heart of that transformation. It stands as a sustainable model for moving potential therapies from bench to bedside, and for training the leaders who will replicate its success elsewhere. The Center is also the flagship of FAST’s Pillar 4 strategy, which focuses on building the systems and expertise needed to deliver advanced therapies. Alongside efforts like biomarker development, newborn screening, and the global AS registry, the Center serves as both a state-of-the-art site for complex neurogenetic trials and a training ground for physicians who will carry this model back to institutions around the world.
“This Center represents the culmination of years of focused investment and a deep belief in what’s possible when we remove barriers to progress,” said Alana Newhouse, president of FAST. “It’s not the end of the story; it’s a beginning.”
By transforming trial readiness for the Angelman syndrome community, FAST is setting a standard that can inform rare disease research globally. This is the future of rare disease research: coordinated, efficient, and centered on families.
Invest in What Comes Next
FAST’s Pillar 4 strategies are designed to remove the barriers that slow promising therapies from reaching the clinic. This means creating the systems, training the experts, and building the infrastructure rare disease trials need to succeed. Your support fuels this work and ensures we are ready when the next breakthrough emerges.
Donate now to help accelerate the next wave of progress.