Plain Language Summary of the ASCEND study looking at the results of treatment with olipudase alfa after one year in adults with acid sphingomyelinase deficiency (ASMD)

Keyphrases

• Acid Sphingomyelinase 20%
• Acid Sphingomyelinase Deficiency 100%
• Blood Cells 10%
• Bloodstream 10%
• Cholesterol 10%
• Clinical Trial Registry 10%
• ClinicalTrials.gov 10%
• Clot 10%
• Enzyme Replacement Therapy 10%
• Fatty Substance 10%
• Headache 10%
• Inhaled Air 10%
• Lipids 10%
• Liver 40%
• Liver Cells 10%
• Liver Enzymes 10%
• Liver Function 10%
• Liver Size 10%
• Lung 50%
• Lung Function 10%
• Lung Images 10%
• Olipudase Alfa 100%
• Placebo 20%
• Plain Language Summary 100%
• Platelets 10%
• Poor Health 10%
• Rare Genetic Disease 10%
• Safety Concerns 10%
• Sphingomyelin 60%
• Spleen 30%
• Spleen Size 10%
• Stop the Bleed 10%
• Treatment Results 100%
• Treatment Side Effects 10%
• Well Treatment 10%

Pharmacology, Toxicology and Pharmaceutical Science

• Clinical Trial 10%
• Diseases 20%
• Headache 10%
• Liver Enzyme 10%
• Olipudase Alfa 100%
• Placebo 20%
• Side Effect 40%
• Sphingomyelin 60%
• Sphingomyelin Phosphodiesterase 100%