Lysosomal Storage Disease Research

The Maegawa lab focuses on developing therapies for lysosomal storage diseases (LSDs) based on critical pathogenic mechanisms underlying the neurological manifestations of these disorders. The Maegawa team works with clinicians to discover efficacious and robust therapies, including small molecules and delivery tools across the blood-brain barrier (BBB), as well as discover biomarkers to stage disease and monitor therapeutic responses together with basic scientists in defining mechanisms of disease progression. The Maegawa lab is now located at Columbia University Irving Medical Center, Pediatric Research Branch.