Technology Overview

The invention proposes a new approach to rare genetic diseases for which no cures are available, only symptomatic treatments through the preparation of novel multivalent molecules active toward the human enzymes responsible for them. Lysosomal storage diseases (LSDs) are a group of rare metabolic diseases caused by deficits in lysosomal enzymes to date orphaned by treatments

The invention is aimed at developing an effective, noninvasive therapy for the treatment of two metabolic diseases, Mucopolysaccharidosis IV (also known as Morquio A) and Mucopolysaccharidosis II (also known as Hunter’s disease). Lysosomal storage diseases, a group of inherited metabolic disorders, are rare when taken individually, but are substantially numerous when taken as a whole since about one in 5000-8000 infants is