Method of treatment of a mucopolysaccharidosis

#The present invention relates to the use of odiparcil, or of a pharmaceutical composition containing this compound, in the treatment of a mucopolysaccharidosis.

#Mucopolysaccharidoses (MPSs) are degenerative genetic diseases linked to an enzymatic defect. In particular, MPSs are caused by the deficiency or the inactivity of lysosomal enzymes which catalyze the gradual metabolism of complex sugar molecules called glycosaminoglycans (GAGs). These enzymatic deficiencies cause an accumulation of GAGs in the cells, the tissues and, in particular, the cell lysosomes of affected subjects, leading to permanent and progressive cell damage which affects the appearance, the physical capacities, the organ function and, in most cases, the mental development of affected subjects.

#Eleven distinct enzymatic defects have been identified, corresponding to seven distinct clinical categories of MPS. Each MPS is characterized by a deficiency or inactivity of one or more enzymes which degrade mucopolysaccharides, namely heparan sulfate, dermatan sulfate, chondroitin sulfate and keratan sulfate.

#FIG. 1 shows the effect of odiparcil on GAG levels in bovine aortic endothelial cells.

#FIG. 2 shows the effect of odiparcil on GAG synthesis by normal human dermal fibroblasts.

#FIG. 3 shows the effect of odiparcil on intracellular chondroitin sulfate content in fibroblasts from MPS I patients.

#According to a first aspect, the invention relates to a method of treatment of a mucopolysaccharidosis type I or H, which comprises administering to a subject in need thereof a therapeutically effective amount of odiparcil.

#Odiparcil and the process for obtaining it are described in patent application EP-A-0 421 829.

#In the context of the present invention, the term “odiparcil” denotes the “β-D-xylopyranoside” form.