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

gan Hogan, Melissa J., Stephens, Kim, Smith, Erin, Jalazo, Elizabeth R., Hendriksz, Christian J., Edwards, Lloyd J., Bjoraker, Kendra J.
Cyhoeddwyd yn Mol Genet Metab Rep (2020)