METHODS AND COMPOSITIONS FOR TREATING LYSOSOMAL STORAGE DISORDERS: AMBROXOL AS TREATMENT AGENT FOR MUCOPOLYSACCHARIDOSES III (SANFILIPPO SYNDROME)

摘要

Methods for treating MPS III diseases are disclosed. The methods involve administering a therapeutically effective amount of a composition comprising ambroxol to a subject with the disease. Also disclosed are pharmaceuticals suitable for treating MPS III diseases.