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Phase I and II clinical trials for the mucopolysaccharidoses

25 Oct 2017

Abstract excerpt

INTRODUCTION: The mucopolysaccharidoses are lysosomal diseases characterized by deficient activity of one of the enzymes that degrades glycosaminoglycans. Treatment options are limited; therefore, new treatments are under investigation. Areas covered: We review the medicinal products for the treatment of mucopolysaccharidoses that are currently being investigated in phase I and phase II clinical trials. Expert...

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PMID: 29065735
DOI: 10.1080/13543784.2017.1397130

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