This CPB has been revised to state that the following are considered experimental and investigational: (i) intrathecal idursulfase for progressive cognitive impairment in individuals with Hunter syndrome (mucopolysaccharidosis II), and (ii) gene therapy and pharmacological chaperone therapy (also known as enzyme-enhancement therapy) for the treatment of mucopolysaccharidoses.

The title of this CPB has also been changed to “Lysosomal Storage Disorders: Treatments”.