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ERT in Children with Mucopolysaccharidosis
NCT05006222Completed
- Sponsor
- Hasan Kalyoncu University
- Start
- Feb 2020
- Primary completion
- Mar 2021
- Countries
- 1
01
Overview
Open-label trial in Mucopolysaccharidosis (MPS) (2 arms), sponsored by Hasan Kalyoncu University. Primary endpoints: Pediatric Quality of Life Inventory at 1 year, Functional Independence Measure at 1 year and 1 more. Started Feb 2020; status: completed.
02
Conditions
Mucopolysaccharidosis (MPS)
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