Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy

Medicine & Life Sciences

• Type A Niemann-Pick Disease 100%
• Enzyme Replacement Therapy 77%
• Acids 44%
• Clinical Trials 38%
• Splenomegaly 22%
• Lung Volume Measurements 17%
• Niemann-Pick Diseases 10%
• Lipids 8%
• Carbon Monoxide 6%
• Mortality 6%
• Natural History 6%
• Liver Cirrhosis 6%
• Lung Diseases 5%
• Spleen 5%
• Observational Studies 5%
• Health Care Outcome Assessment 4%
• Biomarkers 4%
• Hemorrhage 4%
• Quality of Life 4%
• Morbidity 3%
• Lung 3%

Chemical Compounds

• Acid 20%
• Lipid 10%
• Marker 6%
• Carbon Monoxide 6%
• Disorder 6%
• Diffusion 4%
• Volume 4%