Niemann-pick Disease Types and
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CLINICAL_TRIALS research · 2025-09-12Clinical Trial Update: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
This clinical trial update describes ScreenPlus, a comprehensive, multi-disorder newborn screening program that is currently enrolling by in…
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CLINICAL_TRIALS trial_result · 2024-07-03Clinical Trial Update: A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency [Phase 2]
This document reports long-term results from a Phase 2 study of olipudase alfa in patients with acid sphingomyelinase deficiency (Niemann-Pi…