Webinar Details
Title: BiomarkersA measurable sign in the body that helps track disease progression or treatment response. of Central Nervous System Involvement in Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart.: New Learnings
Date: Thursday, September 24, 2026
Time: 10 a.m. CT / 11 a.m. ET
Speaker:
Kristen Hagarty-Waite, PhD, RDN, LDN
Webinar Overview:
Central nervous system (CNS) manifestations persist in patients with Pompe disease, despite enzyme replacement therapy, as current formulations cannot cross the blood brain barrier. Plasma glial fibrillary acidic protein (GFAP) and neurofilament light chain (NfL) have emerged as biomarkers to monitor CNS involvement in Pompe disease, particularly for those with the infantile-onset phenotype. In this presentation, we will share our learnings, so far, regarding biomarkers of CNS involvement.
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Speaker Biography
Kristen Hagarty-Waite, PhD, RDN, LDN
Postdoctoral Associate,
Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center
Dr. Hagarty-Waite is a registered dietitian with over 7 years of experience in the acute care setting. She completed her PhD in Nutrition from the University of North Carolina at Greensboro in the Spring of 2025, during which she studied the impact of diet and genetics on brain iron metabolism, neuroinflammation, and dopamine kinetics. She joined the Department of Pediatrics, Division of Medical Genetics at Duke University in July of 2025 where she is researching the central nervous system manifestations of Pompe disease.