By: Grace Huff
At the Norman Fixel Institute for Neurological Diseases at UF Health, hope is not just a concept, it’s something being actively built every day for individuals and families living with Huntington’s disease (HD). This rare, inherited neurodegenerative disorder slowly breaks down nerve cells in the brain, affecting movement, thinking, and mood. While there is currently no cure, the work being done at Fixel is reshaping what’s possible for patients today and in the future.
Huntington’s disease is caused by a mutation in the HTT gene that leads to abnormal protein buildup in the brain. Symptoms typically appear between ages 30 and 50, though in some cases they can develop earlier as juvenile-onset HD. Individuals often experience a combination of uncontrolled, dance-like movements known as chorea, emotional disturbances such as depression or irritability, and cognitive decline that worsens over time. As the disease progresses, so too does the need for comprehensive care, something the Fixel Institute is uniquely equipped to provide.
The UF Health Huntington’s Disease Clinic has been designated a Center of Excellence by the Huntington’s Disease Society of America (HDSA), an honor awarded to only 50 centers nationwide. This distinction reflects the depth and quality of care provided at Fixel, as well as its commitment to advancing research and supporting families through every phase of the disease. “Being recognized as an HDSA Center of Excellence reflects our commitment to providing the highest standard of care, research, and support to families affected by Huntington’s disease,” said Nikolaus McFarland, MD, PhD, director of the UF HD Clinic, “It’s both an honor and a responsibility we take seriously.”
That responsibility comes to life through a collaborative, multidisciplinary care model that brings together neurologists, psychiatrists, neuropsychologists, genetic counselors, rehab specialists, dietitians, social workers, and research coordinators. “Huntington disease affects not just the patient, but also their family and caregivers,” said McFarland. “The complexity of HD requires a comprehensive, multidisciplinary approach. Our team works together to support the whole patient and their family.”
Serving more than 80 families each year, the UF HD Clinic continues to grow in both reach and capability. Over the past year, the team added a second genetic counselor as well as a dedicated case manager and a Center of Excellence clinic and research coordinator to enhance patient services and outreach efforts. The team is also deeply involved in training the next generation of HD care providers, including neurology residents and fellows, several of whom have gone on to start HD clinics of their own.
In addition to compassionate clinical care, the Fixel Institute is a driving force in Huntington’s research and experimental therapeutics. As an active site for the global ENROLL-HD registry trial, the team contributes to a growing body of knowledge that shapes future therapies. Fixel also played a key role in the KINECT-HD2 trial, which helped lead to the FDA approval of valbenazine (Ingrezza) for treatment of HD chorea. Ongoing studies such as PROOF-HD and the gene therapy-focused Generation HD2 trial aim to address not only symptoms, but the underlying biology of the disease. “At Fixel, we’re not only treating patients, but we’re also pushing forward the science,” said McFarland. “We’re actively involved in clinical trials and basic research aimed at understanding the disease and developing meaningful new therapies.”
Throughout May, which marks Huntington’s Disease Awareness Month, the clinic reflects on the impact of its mission and the patients who inspire it. “Awareness is just the beginning,” said McFarland. “This month, we’re reminded of how vital our work is to the patients and families we serve and who inspire us to keep building a future where Huntington’s disease is no longer a life-limiting diagnosis.”
The future of HD care is already being reimagined at Fixel. From exploring genetic modifiers that influence disease progression to adopting new HD staging systems for research and trials, the institute is laying the foundation for more personalized, effective care. “The future for HD patients appears brighter each year with the advances in our understanding of the disease,” McFarland added. “We believe the dream of a day without Huntington’s disease is not only possible, it’s getting closer.”
As an HDSA Center of Excellence, the Fixel Institute continues to lead with compassion, collaboration, and discovery—investing in hope and working toward a world where HD is no longer a diagnosis, but a disease of the past.
What’s Next: A Brighter Horizon
Looking ahead, the team at Fixel is excited about the future of HD research and care. Breakthroughs such as a new staging system for clinical trials and the discovery of genetic modifiers that influence disease onset are fueling next-generation treatment approaches.
“The future for HD patients appears brighter each year with the advances in our understanding of the disease,” Dr. McFarland said. “We believe the dream of a day without Huntington’s disease is not only possible, it’s getting closer.”
From bold research to compassionate care, the UF Fixel Institute continues to redefine what it means to live well with HD, and what it will mean to live in a world beyond it