Ensayos clínicos

Leukodystrophy

Displaying 2 studies

  • 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).

  • Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C Rochester, MN

    A prospective, randomised, double-blind, placebo controlled therapeutic study in patients with confirmed diagnosis of NiemannPick disease type C (NPC). The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the patient's current prescribed best standard of care; patient's standard of care may, or may not, include miglustat.

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