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A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy

Approved WMORecruitment stopped

The primary objective of this study is to evaluate the effects of intrathecal (IT) administration of SHP611 (also known as TAK-611) on the time to loss of locomotion, as indicated by category 5 or higher in the Gross Motor Function Classification in…

Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome (NEPTUNE)

Approved WMORecruitment stopped

All efficacy study objectives will be assessed in the pediatric AS study population of subjects who are 4 to 12 years old. All safety study objectives will be assessed in the pediatric AS study population of subjects who are 2 to 12 years old.…

10-year extension study: prospective systematic follow-up for X-linked adrenoleukodystrophy.

Approved WMORecruiting

The primary objective of the study is to develop new sensitive and quantitative outcome measures for clinical trials in ALD. The secondary objective is to identify biomarkers to stratify patients based on disease progression rate.