Lysosomal Storage Disease Program

Our Mission

The UF Lysosomal Storage Disease Program is committed to offering comprehensive and personalized clinical care for patients suffering from Lysosomal Storage Diseases (LSDs). LSDs are genetic disorders that are caused by specific enzyme deficiencies resulting in multi-systemic disease, presenting with neurological, renal, cardiovascular, gastrointestinal, musculoskeletal, ophthalmological, and respiratory problems. The LSD program provides diagnosis, management, treatment, and monitoring of several different treatments available for patients affected by LSD including enzyme replacement therapy, small molecule therapy, hematopoietic stem cell therapy, and others.

UF clinical LSD program unites outstanding physicians from multiple specialties into a cooperative team devoted to offering excellence in patient care. S. Parrish Winesett, MD coordinates the multi-disciplinary group from primary specialists in their respective sub-specialties. Our team has experience and resources to treat a wide range of complications commonly seen in these inherited metabolic diseases.

Based on the UF tradition of integrating clinical and basic research, teaching, and patient care, the LSD program is also involved in several clinical studies including clinical trials with the most advanced therapeutic agents. The unique characteristics of the program are an integrated approach to patients suffering from multi-systemic LSDs following UF principles of the pursuit of excellence and innovation in patient care.

The UF Lysosomal Storage Disease Program is directed by Parrish Winesett, MD physician-scientist from the Division of Neurology in the Department of Pediatrics, College of Medicine, University of Florida. Dr. Winesett aims to understand the molecular mechanisms of LSD and develop new therapeutic approaches. Dr. Winesett coordinates the necessary multi-disciplinary and personalized for affected patients and their families. Dr. Winesett also collaborates with LSD centers throughout the United States and around the world to bring advances from basic research to clinical care.