Research
Dr. Polgreen’s research focuses on ways to improve the treatment and understanding of the mucopolysaccharidoses (MPS) and osteopetrosis. She has previously designed and implemented multiple I-IND studies in MPS disorders (NCT04018755; NCT00748969; NCT03153319; NCT02437253), most recently a clinical trial of anakinra in 23 participants with MPS III that was published in Nature Medicine.
Dr. Polgreen has a proven record of recruiting patients with MPS for both clinical trials and natural history studies. Her experience in natural history studies includes being the leader on a 10-year, observational study of musculoskeletal disease in MPS disorders that was a multi-site study of 55 participants aimed at quantifying disease progression and identifying outcome measures for future clinical trials, funded through the Lysosomal Disease Network U54 contract.
She has also designed and managed an R01 funded osteopetrosis REDCap-based registry that is used to collect population-based natural history data from individuals with osteopetrosis. Finally, she is involved with the MPS community through membership in the National MPS Society Scientific Advisory Board and a member of multiple independent monitoring committees for industry-sponsored clinical trials.
Biography
Dr. Lynda Polgreen is the Clinical Director of the Center for Treatment of Rare Disease (CTRD) and the Associate Director of the Institute for Translational Genomics and Population Sciences. Clinically, she sees Pediatric Endocrinology patients at Harbor-UCLA and participates in a multi-disciplinary clinic for patients with MPS at Children’s Hospital Orange County.
She has experience leading both investigator-sponsored and industry-sponsored clinical trials in MPS and other rare pediatric diseases, as well as longitudinal natural history studies in MPS. She has previously designed and implemented multiple I-IND studies in MPS disorders (NCT04018755; NCT00748969; NCT03153319; NCT02437253), most recently a clinical trial of anakinra in 23 participants with MPS III that was published in Nature Medicine.
Additionally, she sees patients with all MPS disorders in a multi-disciplinary clinic at Children’s Hospital Orange County. Combining her research and clinical experience, she has cared for over 100 individuals with MPS over the last 20 years. From these experiences, Dr. Polgreen has developed a “hands on” understanding of the clinical course in MPS and the areas where improved treatment is urgently needed – Sanfilippo syndrome is one of these areas as there is currently no therapy approved for treatment or palliation of MPS III.