Harbor-UCLA Medical Center

  • Nephrotic Syndrome Rare Disease Clinical Research Network III (NEPTUNE)

    Focal and Segmental Glomerulosclerosis, Minimal Change Disease and Membranous Nephropathy, presenting as Nephrotic Syndrome (NS), are a group of rare renal diseases that may cause serious complications and end-stage kidney disease, generating significant individual, societal and economic burdens. The Nephrotic Syndrome Study Network (NEPTUNE) brings together physician scientists at 26 sites in the

  • Lysosomal Disease Network (LDN)

    Lysosomal disorders (LD) are a group of approximately 70 inherited metabolic conditions resulting from defects in lysosomal function; usually deficiency of a single enzyme required for the metabolism of lipids, glycoproteins, or mucopolysaccharides. Collectively, LD are not especially rare; estimates suggest that approximately 1 in 5,000 newborns will be affected with one identified LD