A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT.
Karen S YeeCostel ChirilaEric DavenportDeirdre MladsiChristine BarnettWilliam G KronenbergerPublished in: Orphanet journal of rare diseases (2023)
ClinicalTrials.gov NCT02055118 (registration date: 4 February 2014) and NCT02412787 (registration date: 9 April 2015).