Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency

Medicine & Life Sciences

• olipudase alfa 100%
• Type A Niemann-Pick Disease 73%
• Acids 32%
• Sphingomyelins 20%
• Niemann-Pick Diseases 17%
• chitotriosidase 8%
• Sphingomyelin Phosphodiesterase 7%
• Enzyme Replacement Therapy 7%
• Proof of Concept Study 6%
• Acute-Phase Reaction 6%
• Investigational Therapies 6%
• Ceramides 6%
• Safety 6%
• Arthralgia 5%
• Platelet Count 5%
• Lung Diseases 4%
• Abdominal Pain 4%
• Half-Life 4%
• Nausea 4%
• Headache 4%
• Hypersensitivity 4%
• Spleen 4%
• Pharmacokinetics 4%
• Poisons 4%
• Lipids 3%
• Quality of Life 3%
• Antibodies 3%
• Liver 2%
• Serum 2%

Chemical Compounds

• Dose 33%
• Sphingomyelin 23%
• Acid 15%
• Safety 10%
• Ceramide 7%
• Reaction Half Life 5%
• Reduction 5%
• Pharmacokinetics 4%
• Toxic 4%
• Metabolite 4%
• Lipid 4%
• Plasma 3%
• Volume 3%
• Time 2%