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Human Acid Sphingomyelinase Deficiency
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Human Acid Sphingomyelinase Deficiency trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Human Acid Sphingomyelinase Deficiency trials you may qualify forTo evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of rhASM in adult patients with Acid Sphingomyelinase Deficiency (ASMD) follo…
The purpose of this study is to determine the safe range of single doses of rhASM administered to adults with ASM deficiency.
The main source of energy for the brain comes from a combination of oxygen and glucose (sugar). For brain cells to function normally they must receive a constan…