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          Last Updated: 10/31/2025

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          1 clinical trial found

            A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age

            A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age

            Enrollment Status: Recruiting
            Publish Date: May 16, 2025
            Intervention Type: Drug

            Summary: The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis. The main questions it aims to answer are: * study the effect of velmanase alfa on a marker of the disease called GlcNAc(Man)2 after one year of therapy * explore how the child's body reacts to velmanase alfa during the therapy The ...

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            Last Updated: 10/31/2025