LAU, HEATHER

Dr. Heather A. Lau is a board-certified neurologist and clinical researcher, specializing in neurogenetic disorders, lysosomal storage diseases, leukodystrophies, and Gaucher disease. She has an extensive background in neurology, rare disease research, and pharmaceutical development, contributing to innovative therapeutic strategies for patients with inherited nervous system disorders.

Dr. Lau previously served as Director of the Division of Neurogenetics at NYU Langone Health, where she led clinical care and research initiatives focused on rare neurogenetic conditions. She maintained a large practice treating children and adults, integrating cutting-edge therapies into patient care and clinical trials. She has served as principal investigator for over 40 clinical trials, evaluating the efficacy of small molecules, chaperone therapy, substrate reduction therapy, enzyme replacement therapy, and gene therapy in treating neurogenetic disorders.

Dr. Lau now serves as Executive Director of Global Clinical Development at Ultragenyx, leading biopharmaceutical efforts to develop novel therapies for neurological diseases. Additionally, she is an Adjunct Assistant Professor at Yale University, contributing to the Lysosomal Program in the Department of Internal Medicine, where she collaborates on research focused on Gaucher disease and other metabolic disorders.

Her work integrates clinical neurology with pharmaceutical development, ensuring that patients with rare diseases have access to groundbreaking treatments. She has contributed extensively to scientific literature, publishing studies on lysosomal storage disorders, ataxias, and neurodevelopmental diseases. Dr. Lau collaborates with international research teams, refining diagnostic tools and therapeutic approaches for genetic neurological conditions.

Dr. Lau earned her Bachelor of Science with honors from Cornell University, followed by a Master of Science in Biochemistry and Molecular Biology from New York Medical College. She completed her MD at the University of Rochester, training in pediatrics at Montefiore Medical Center and child neurology at NYU School of Medicine, where she also pursued fellowship training in neurogenetics.

Beyond her research and clinical work, Dr. Lau is actively involved in medical education and mentorship, training healthcare professionals in neurogenetics and pediatric neurology. Her contributions to neurogenetic research, rare disease treatment, and medical education continue to shape the field, improving diagnostic accuracy and therapeutic strategies for patients with complex neurological conditions.

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