A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy
Rochester, MN
The primary purpose of this study is to evaluate the effects of intrathecal (IT) administration of SHP611 on gross motor function, using the Gross Motor Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with metachromatic leukodystrophy (MLD).