Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase

Publication date: December 2020Source: Molecular Genetics and Metabolism Reports, Volume 25Author(s): Melissa J. Hogan, Kim Stephens, Erin Smith, Elizabeth R. Jalazo, Christian J. Hendriksz, Lloyd J. Edwards, Kendra J. Bjoraker

Source: Molecular Genetics and Metabolism Reports - October 22, 2020 Category: Genetics & Stem Cells Source Type: research