New treatments for the mucopolysaccharidoses: from pathophysiology to therapy
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DOI: 10.1186/s13052-018-0564-z
Abstract: Enzyme replacement therapy is currently considered the standard of care for the treatment of mucopolysaccharidoses (MPS) type I, II, VI, and IV. This approach has shown substantial efficacy mainly on somatic symptoms of the patients,… read more here.
Keywords: treatments mucopolysaccharidoses; pathophysiology therapy; mucopolysaccharidoses pathophysiology; biology ... See more keywords