Not applicableUnknownNCT05904366What this trial is testingMagnetic Resonance Spectroscopy in Acid Sphingomyelinase DeficiencyWho this might be right forAsmd, Visceral Type Eline C. B. Eskes 34
Not applicableEnrolling By InvitationNCT05368038What this trial is testingScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramWho this might be right forAcid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more Albert Einstein College of Medicine 100,000
Large-scale testing (Phase 3)Study completedNCT02004691What this trial is testingEfficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase DeficiencyWho this might be right forSphingomyelin Lipidosis Genzyme, a Sanofi Company 36